New Drug Approvals

Home » Posts tagged 'USA'

Tag Archives: USA

Advertisements
DRUG APPROVALS BY DR ANTHONY MELVIN CRASTO .....FOR BLOG HOME CLICK HERE

Blog Stats

  • 2,623,488 hits

Flag and hits

Flag Counter

Enter your email address to follow this blog and receive notifications of new posts by email.

Join 2,416 other followers

Follow New Drug Approvals on WordPress.com

Categories

Flag Counter

ORGANIC SPECTROSCOPY

Read all about Organic Spectroscopy on ORGANIC SPECTROSCOPY INTERNATIONAL 

Enter your email address to follow this blog and receive notifications of new posts by email.

Join 2,416 other followers

DR ANTHONY MELVIN CRASTO Ph.D

DR ANTHONY MELVIN CRASTO Ph.D

DR ANTHONY MELVIN CRASTO, Born in Mumbai in 1964 and graduated from Mumbai University, Completed his Ph.D from ICT, 1991,Matunga, Mumbai, India, in Organic Chemistry, The thesis topic was Synthesis of Novel Pyrethroid Analogues, Currently he is working with GLENMARK PHARMACEUTICALS LTD, Research Centre as Principal Scientist, Process Research (bulk actives) at Mahape, Navi Mumbai, India. Total Industry exp 30 plus yrs, Prior to joining Glenmark, he has worked with major multinationals like Hoechst Marion Roussel, now Sanofi, Searle India Ltd, now RPG lifesciences, etc. He has worked with notable scientists like Dr K Nagarajan, Dr Ralph Stapel, Prof S Seshadri, Dr T.V. Radhakrishnan and Dr B. K. Kulkarni, etc, He did custom synthesis for major multinationals in his career like BASF, Novartis, Sanofi, etc., He has worked in Discovery, Natural products, Bulk drugs, Generics, Intermediates, Fine chemicals, Neutraceuticals, GMP, Scaleups, etc, he is now helping millions, has 9 million plus hits on Google on all Organic chemistry websites. His friends call him Open superstar worlddrugtracker. His New Drug Approvals, Green Chemistry International, All about drugs, Eurekamoments, Organic spectroscopy international, etc in organic chemistry are some most read blogs He has hands on experience in initiation and developing novel routes for drug molecules and implementation them on commercial scale over a 30 year tenure till date Dec 2017, Around 35 plus products in his career. He has good knowledge of IPM, GMP, Regulatory aspects, he has several International patents published worldwide . He has good proficiency in Technology transfer, Spectroscopy, Stereochemistry, Synthesis, Polymorphism etc., He suffered a paralytic stroke/ Acute Transverse mylitis in Dec 2007 and is 90 %Paralysed, He is bound to a wheelchair, this seems to have injected feul in him to help chemists all around the world, he is more active than before and is pushing boundaries, He has 9 million plus hits on Google, 2.5 lakh plus connections on all networking sites, 50 Lakh plus views on dozen plus blogs, He makes himself available to all, contact him on +91 9323115463, email amcrasto@gmail.com, Twitter, @amcrasto , He lives and will die for his family, 90% paralysis cannot kill his soul., Notably he has 19 lakh plus views on New Drug Approvals Blog in 216 countries......https://newdrugapprovals.wordpress.com/ , He appreciates the help he gets from one and all, Friends, Family, Glenmark, Readers, Wellwishers, Doctors, Drug authorities, His Contacts, Physiotherapist, etc

Personal Links

Verified Services

View Full Profile →

Categories

Flag Counter
Advertisements

USA Viewership touched 3 lakhs on New Drug Approvals


str0

USA Viewership touched 3 lakhs on New Drug Approvals

https://newdrugapprovals.org/

Total 16.9 lakhs in 213 countries

Advertisements

Scaling up from mg to Kgs – Making your First GMP Batch


STR1

Scaling up from mg to Kgs – Making your First GMP Batch 

6th – 7th October 2016, Clearwater, USA

the course was very informative and it allowed me to see the big picture from discovery stage to pilot plant” 
Genentech

Course Outline:

  • Introduction
  • Making the first 100g non-GMP Batch
  • Non-GMP vs GMP preparation
  • Physical version and form
  • Process safety and raw materials supply
  • Scaling into fixed vessels
  • Technology transfer
  • Genotoxic impurities
  • Case studies and Review

Who should attend:

  • Project managers
  • Project leaders
  • Bench chemists
  • New starters
  • MedChem Support teams

This course aims to provide attendees with a good understanding of the issues involved taking development candidates to the first in human trials.

Click here to Download the Course Brochure

Presented by Dr John Knight, JKonsult Ltd

John Knight

Managing Director at JKONSULT Ltd

STR1
Click here to Download the Course Brochure

“Brilliant Course, learn lots of tips and tricks”
Vertex

First incursion into Chemical Development has been very, very educational. John’s way of explaining the material has been wonderful.”
Almirall

Very clear and interesting sessions with a lot of relevant examples and not only theory.” 
Oribase Pharma
LINK
LITERATURE FROM INTERNET ON HIS TOPIC
//////////Scaling up,  mg to Kgs, Making,  First GMP Batch, SCIENTIFIC UPDATE,  JOHN KNIGHT, Clearwater, USA

deleted


deleted

CARMEN DRAHL….Tribute to a Great Writer


CARMEN DRAHL

Award-winning science communicator and social media power user based in Washington, DC.

Carmen Drahl is a multimedia science journalist and chemistry communicator based in Washington, DC.

A social media evangelist, Carmen started her first chemistry blog in 2006. Today, she regularly leverages Twitter, Facebook, and Google Plus Hangouts in her reporting.

Carmen has written about how life may have originated on Earth, explained how new medications get their names, and covered the ongoing issues plaguing the forensic science community. Her video on the food science behind 3D printed cocktail garnishes won the 2014 Folio Eddie Award for Best Association Video.

Until December 2014, Carmen worked at Chemical & Engineering News magazine. Her work has also been featured at Scientific American’s blog network, SiriusXM’s Doctor Radio, and elsewhere.

Carmen holds a Ph.D. in chemistry from Princeton University.

 

Specialties:
interviewing, science writing, social media, Twitter, Storify, YouTube,
public speaking, hosting, video production, iPhone videography,
non-linear video editing, blogging (WordPress and Blogger), HTML website
coding

We have been reading her for the past several years and a inspiration for many

Carmen Drahl - Science Communicator

Links

FACEBOOK

https://www.facebook.com/carmenwrites

Carmen Drahl (@carmendrahl) | Twitter

www.linkedin.com/in/carmendrahl/en

http://www.ddn-news.com/

http://cenblog.org/the-safety-zone/

Carmen Drahl – Google+

Carmen Drahl

 

Education

Princeton University

Ph.D., Chemistry

2002 – 2007

Ph.D. with Erik J. SorensenShe was on a team that completed the first total synthesis of
abyssomicin C, a molecule found in small quantities in nature that
showed hints of promise as a potential antibiotic. I constructed
molecular probes from abyssomicin for proteomics studies of its
biological activity.

M.A. with George L. McLendon

worked
toward developing a drug conjugate as a potential treatment for cancer. I
synthesized a photosensitizer dye-peptide conjugate for targeting the
cell death pathway called apoptosis.

image

At a reception before the Alumni Day luncheon, President Tilghman (third
from left) congratulated the winners of the University’s highest awards
for students: (from left) Pyne Prize winners Lester Mackey and Alisha
Holland; and Jacobus Fellowship recipients Sarah Pourciau, Egemen
Kolemen and Carmen Drahl. Unable to attend the event was Jacobus Fellowship winner William Slauter. (photo: Denise Applewhite

B.A., Chemistry

1998 – 2002

Graduated
summa cum laude with specialized honors in chemistry. Honors thesis
entitled “Structural, kinetic, and mechanistic studies: the protein
tyrosine phosphatases CD45 and PTP1B”
Activities and Societies: Phi Beta Kappa

  Carmen Drahl, Class of 2002,

 

Experience

Science Journalist

Freelance

January 2014 – Present Washington D.C. Metro Area

Multimedia
science journalist – I deliver clean products on time. Experience in
reporting on chemistry, food science, history of science, drug
development, science education.

Senior Editor, Chemical & Engineering News

American Chemical Society

August 2007 – December 2014 (7 years 5 months)Washington D.C. Metro Area

Reporting:Cover the science of chemistry for C&EN, the American Chemical
Society’s weekly magazine, circulation 160,000. Track new research
findings daily, particularly in forensic science, drug discovery,
organic chemistry, and food science.

Video:

Doubled circulation to C&EN’s YouTube channel in 2013. Scripted, narrated, edited footage.

Managed a core team of 4 and collaborated with other reporters to
produce 30 videos, some reproduced in The Atlantic, Scientific American,
Eater National, The Daily Mail.

Incepted, scripted, and co-hosted
“Speaking of Chemistry”, a monthly web show that summarizes top
chemistry news for the busy scientist.

Social Media:

Developed magazine-wide best practices for YouTube videos and Twitter. Ran staff workshops about Storify, Slashdot, and Reddit.

Hosting/Public Speaking:

Topics include communicating chemistry simply, transitioning from a
Ph.D. to careers in science communication. Moderated discussions on
chemophobia, social media usage in the chemical sciences. On-camera
co-host for web newscasts produced by ACS.

Innovation:

With
C&EN art and web teams, developed first-for-the-magazine features,
including a 90th anniversary commemorative timeline poster, a pullout
guide to top conference speakers, interactive quizzes and database
searches.

Carmen Drahl, senior editor of Chemical and Engineering News,
used her Ph.D. in chemistry as a springboard into the career she
envisioned for herself. Here she shares some advice that helped her make
the decision.

Carmen Drahl made the transition to a writing
career while earning a Ph.D. in chemistry at Princeton University. Born
and raised in New Jersey, she now lives in Washington, D.C., and reports
for Chemical and Engineering News (C&EN). At C&EN
she has written about how new medications get their names, explained
the science behind a controversial hair-straightening product, and
covered the scientific firestorm sparked by an alleged arsenic life
form. Her work has been featured on SiriusXM’s Doctor Radio, Radio New Zealand’s This Way Up, and elsewhere. Her coverage has also been recognized by MIT’s Knight Science Journalism Tracker.

(Open)1 honor or award
Scientific Cocktails: Award-winning video
Scientific Cocktails: Award-winning video
Speaking of Chemistry: All About Tinsel
Speaking of Chemistry: All About Tinsel

Carmen Drahl

Twitter Maven

World Central Kitchen

March 2013 – August 2014 (1 year 6 months)Washington D.C. Metro Area

she was the “voice of Twitter” for World Central Kitchen, the humanitarian
organization founded by renowned Chef José Andrés. Doubled followers to
Twitter account in 2013, developed Twitter strategy for projects and
events. Edited Annual Report, press releases and other communication
materials. Volunteered in person at outreach events.

Contributing Editor, AWIS Magazine

Association of Women in Science

December 2005 – August 2007 (1 year 9 months)

sHE
reported and wrote profiles of prominent women scientists in a range of
fields (molecular biology, physics, geoscience) for the Research
Advances column in AWIS Magazine.

Writer, various publications

Princeton University

April 2005 – May 2007 (2 years 2 months)

She
reported and wrote news for the Princeton University News Office’s
Research Notes, and wrote news and features for the Princeton University
Chemistry Department’s Industrial Affiliates Program Newsletter and
Chemistry Alumni Newsletter.

Honors & Awards

Eddie Digital Award- Best Video (B-to-B)

FOLIO Magazine

December 2014

Porter Ogden Jacobus Fellowship

Princeton University

February 2007

NSF Graduate Research Fellowship

National Science Foundation

2002

Volunteer Experience & Causes

Board Member

Princeton Alumni Weekly Magazine

October 2013

Advisory Committee

American Institute of Physics News and Media Services

October 2013

Member, Graduate Alumni Leadership Council

Princeton University

2009 – 2012 (3 years)

INTERVIEW

Continuing with the tradition from last two years, I will occasionally post interviews with some of the participants of the ScienceOnline2010 conference that was held in the Research Triangle Park, NC back in January. See all the interviews in this series here. You can check out previous years’ interviews as well: 2008 and 2009.Today, I asked Carmen Drahl, Associate Editor for Science/Technology/Education at Chemical & Engineering News (find her as @carmendrahl on Twitter) to answer a few questions.Welcome
to A Blog Around The Clock. Would you, please, tell my readers a little
bit more about yourself? Where are you coming from (both geographically
and philosophically)? What is your (scientific) background?
i-b183f89fe33d3d9f0b308a6cb30d9b5b-Carmen Drahl pic1.JPGIt’s a pleasure and a privilege to be interviewed, Bora.Good
conversations make me happy. School was fun for me (well, maybe not
grad school) and that’s evolved into a desire to always be learning
something new. I enjoy doing nothing as much as I enjoy doing things. On
Mondays, if I’m not too busy, I take hip-hop dance classes.her hometown is Hackettstown, New Jersey. M&M’s are made there. I got a
bachelor’s in chemistry from Drew University and a Ph.D. in chemistry at
Princeton. Scientifically my expertise hovers somewhere around the
interface between organic chemistry and biochemistry. A short while
after defending my dissertation, I moved to Washington DC to write for Chemical & Engineering News, and that’s where I’ve been for almost three years now.When and how did you first discover science blogs?Scandal
led me to science blogs. Seriously. In March 2006 I was still an
organic chemistry grad student. Everyone in my lab was buzzing about a
set of retractions in the Journal of the American Chemical Society
(disclosure: today I work for the American Chemical Society, which
publishes JACS). A rising young organic chemistry star retracted the
papers because work by one of his graduate students couldn’t be
reproduced. It was a big deal and became an even bigger deal as the
inevitable rumors (salacious and otherwise) surfaced. The blogosphere
had the details first. So that’s where Google pointed me and the other
members of my lab when we searched for more information. I learned about
the awesome (but sadly now defunct) blogs Tenderbutton and The Endless
Frontier, by Dylan Stiles and Paul Bracher, both chemistry grad students
like me. I also discovered the solid mix of chemistry and pharma at
Derek Lowe’s In the Pipeline, which is still the first blog I visit every day.Tell us a little more about your career trajectory so far: interesting projects past and present?

i-b7bd4d4568d9689c2daf400303c886c3-Carmen Drahl pic2.JPGBy
the time I discovered science blogs I knew my career goals were
changing. I’d already been lucky enough to audit a science writing
course at Princeton taught by Mike Lemonick from TIME, and thought that
maybe science writing was a good choice for me. After reading chemistry
blogs for a while I realized “Hey, I can do this!” and started my own
blog, She Blinded Me with Science, in July 2006. It was the typical grad student blog, a mix of posts about papers I liked and life in the lab.

At C&E News I’ve contributed to its C&ENtral Science
blog, which premiered in spring 2008. I’ve experimented with a few
different kinds of posts- observations and on-the-street interviews when
I run into something chemistry-related in DC, in-depth posts from
meetings, and video demos of iPod apps. One of my favorite things to do
is toy with new audio/video/etc technology for the blog.

What is taking up the most of your time and passion these days? What are your goals?

In March I just started a new era in my web existence- I’m becoming a pharma blogger. I’m the science voice at The Haystack,
C&E News’s new pharma blog and one of seven new blogs the magazine
launched last month. My co-blogger is the talented Lisa Jarvis, who’s
written about the business side of pharma for ten years and who brings a
solid science background to the table as well. I kicked us off by
liveblogging/livetweeting a popular session at the American Chemical
Society’s meeting in San Francisco where drug companies reveal for the
first time the chemical structures of potential new drugs being tested
in clinical trials. The whole thing synced to FriendFeed as well. Folks
followed the talks from all three venues, which was great. I hope I can
continue doing that sort of thing in the future.

For
this August, I’m co-organizing a mini-symposium at the American
Chemical Society meeting in Boston about the chem/pharma blogosphere and
its impact on research and communication. I’m in the process of
inviting speakers right now. It’s my first time doing anything like this
and part of me is petrified that no one will show up. Tips on
organizing a conference session and how not to stress when doing so are
welcome!

More broadly, I’d love to get more chemistry bloggers to
connect with the community that attends ScienceOnline. I don’t ever want
to become that old (or not-so-old) person who is clueless about
them-thar newfangled whosiwhatsits that the kids are using nowadays.

What aspect of science communication and/or particular use of the Web in science interests you the most?

A
few things come to mind, actually. I’d like to think that the web has
made grad school a helluva lot less isolating for science grad students.
You have the virtual journal clubs like Totally Synthetic, posts like SciCurious’s letter to a grad student, etc.

As
a journalist the web’s capacity to equalize fascinates me. I’m
extremely lucky to have a staff gig as a science writer without having
gone to journalism school or landed a media fellowhip and it’s weird to
think that my old blog might’ve helped my visibility. I didn’t know Ed
Yong’s story until Scio10 but I think he’s a highly talented example of
how the web can open doors.

The web’s equalizing power goes to
readers of science content as well as writers, of course. In the ideal
situation a reader can give a writer instant feedback and you can get a
real conversation going, something that was much harder with the
snail-paced system of letters to the editor and reader surveys. Not that
the conversation is always civil. Most of C&EN’s readers have a
decent amount of scientific training, but the debate that rages whenever
we run an editorial about climate change is as intense as any I’ve
seen.

In cases like that I don’t know that the web gives people a
good representation of what the consensus is. For folks who don’t have
scientific training, how do you ensure that people don’t just go to the
content that already confirms their pre-existing beliefs about autism or
global warming? John Timmer touched on this more eloquently in his interview with you,
and I agree with him that I don’t think we have an answer yet. Though
on a slightly different note, I will mention that I’ve been enjoying the
New York Times’s recent attempts to recapture the spontaneity of
flipping through the newspaper in online browsing, like the Times Skimmer for Google Chrome.

What are some of your favourite science blogs? Have you discovered any cool science blogs by the participants at the Conference?

In addition to the blogs I’ve already mentioned I enjoy Carbon-Based Curiosities, Wired Science, Chemistry Blog, and Terra Sigillata, to name a few of the 50 or so blogs on my feed reader.

I discovered scads of new blogs at Scio10 but I’ll focus on the one that’s become required reading for me these days: Obesity Panacea.
I’d covered obesity drug development for C&EN but I’d never met
Travis Saunders and Peter Janiszewski or heard of their blog until the
conference.

What was the best aspect of ScienceOnline2010 for
you? Is there anything that happened at this Conference – a session,
something someone said or did or wrote – that will change the way you
think about science communication, or something that you will take with
you to your job, blog-reading and blog-writing?

Dave Mungeris
my hero – his blogging 102 session was packed with practical tips that I
brought back to C&EN for incorporating into our blogs, such as the
use of the Disqus plugin for catching conversations on social networks,
getting smart about using stats and surveys, etc. Some of that’s already
happened, and some of the ideas are still in the works.

I came
for the nuts-and-bolts blogging tips but I stayed for the conversations,
especially the ones at the bar after the official program was done for
the night. And the icing on the cake was seeing folks I’d worked with
but never met, like Cameron Neylon and you, Bora, and catching up with
people I hadn’t seen in months, like Jean-Claude Bradley, Aaron Rowe,
Jennifer Ouellette and Nancy Shute.

It was so nice to meet you in person and thank you for the interview. I hope to see you again next January.


Company: GlaxoSmithKline

Meant to treat: tumors with loss-of-function in the tumor suppressor
protein PTEN (phosphatase and tensin homolog)- 2nd most inactivated
tumor suppressor after p53- cancers where this is often the case include
prostate and endometrial

Mode of action: inhibitor of
phosphoinositide 3-kinase-beta (PI3K-beta). Several lines of evidence
suggest that proliferation in certain PTEN-deficient tumor cell lines is
driven primarily by PI3K-beta.

Medicinal chemistry tidbits: The GSK
team seemed boxed in because in 3 out of 4 animals used in preclinical
testing, promising drug candidates had high clearance. It turned out
that a carbonyl group that they thought was critical for interacting
with the back pocket of the PI3K-beta enzyme wasn’t so critical after
all. When they realized they could replace the carbonyl with a variety
of functional groups, GSK2636771 eventually emerged. GSK2636771B (shown)
is the tris salt of GSK2636771.

Status in the pipeline: Phase I clinical trials……….http://cenblog.org/the-haystack/2012/03/liveblogging-first-time-disclosures-from-acssandiego/

CARMEN

Posted By on Mar 24, 2012

Phone: 202-872-4502

Fax: 202-872-8727 or -6381


Company: GlaxoSmithKline

Meant to treat: tumors with loss-of-function in the tumor suppressor
protein PTEN (phosphatase and tensin homolog)- 2nd most inactivated
tumor suppressor after p53- cancers where this is often the case include
prostate and endometrial

Mode of action: inhibitor of
phosphoinositide 3-kinase-beta (PI3K-beta). Several lines of evidence
suggest that proliferation in certain PTEN-deficient tumor cell lines is
driven primarily by PI3K-beta.

Medicinal chemistry tidbits: The GSK
team seemed boxed in because in 3 out of 4 animals used in preclinical
testing, promising drug candidates had high clearance. It turned out
that a carbonyl group that they thought was critical for interacting
with the back pocket of the PI3K-beta enzyme wasn’t so critical after
all. When they realized they could replace the carbonyl with a variety
of functional groups, GSK2636771 eventually emerged. GSK2636771B (shown)
is the tris salt of GSK2636771.

Status in the pipeline: Phase I clinical trials……….http://cenblog.org/the-haystack/2012/03/liveblogging-first-time-disclosures-from-acssandiego/

CARMEN

Posted By on Mar 24, 2012

Phone: 202-872-4502

Fax: 202-872-8727 or -6381

  1. Map of washington dc
Washington, D.C.
.
.

.

.

NEW DRUG APPROVALS…….One lakh viewers in USA


Orphan Drugs for Rare Diseases : Top Ten Most Expensive Drugs in USA


Orphan Drugs for Rare Diseases - Top Ten Most Expensive Drugs in the USA
Orphan Drugs for Rare Diseases – Top Ten Most Expensive Drugs in the USA

Drugs are expensive. Thousands of people take drugs that cost from $50,000 to $100,000 per year, such as the cancer drugs Provenge and Avastin and the medication for multiple sclerosis, Lemtrade. But a much smaller number of people have rare conditions that require lifesaving drugs whose prices are astronomical—up to $400,000 per year, prices that may reflect the cost of developing the medication.

A rare or orphan disease is a condition affecting 200,000 or fewer individuals in the United States. Rare diseases once were the neglected stepchild of drug makers, who wanted medicines they could sell to millions of patients. Today, conditions afflicting far smaller numbers are seeing booming interest from the industry. And more companies are taking advantage of grants, tax credits and other incentives of the Orphan Drug Act passed in 1983. In the preceding decade, only 10 drugs for rare diseases had been approved, but more than 400 were approved from 1984 through 2012.

Thinking you pay way too much for your monthly prescriptions? These amazingly expensive drugs may put things into perspective.

Soliris_Eculizumab_$400,000 a year_paroxysmal nocturnal hemoglobinuria_Alexion

Soliris

Soliris has been made famous by Forbes as the world’s single most expensive drug, coming in at $409,500 a year. Soliris is used to treat paroxysmal nocturnal hemoglobinuria, a rare Marchiafava-Micheli blood disease that affects 8,000 Americans.  The med generated $541 million in sales for Alexion Pharmaceuticals in 2010.

Paroxysmal nocturnal hemoglobinuria causes the breakdown of red blood cells and release of hemoglobin into the urine. The sudden, recurring attacks—which are often triggered by stress, exertion or infection—result in anemia. Little is known about the incidence of paroxysmal nocturnal hemoglobinuria, which can affect people of any age, but it’s believed to occur at a rate of about one to five per one million. The drug Soliris, made by Alexion Pharmaceuticals and approved by the Food and Drug Administration in 2007, stops the breakdown of cells. Soliris is a monoclonal antibody—a product engineered in the lab to mimic a natural substance. Studies show it leads to a dramatic improvement of symptoms and reduces the risks of complications.

elaprase_idursulfase-$375,000 a year_Hunter syndrome_Shire

Elaprase

Patients who suffer from Hunter syndrome, an inherited disease caused by a lack of the enzyme iduronate sulfatase can find relief in the recombinant form of this enzyme, but at an incredibly high price of $375,000 each year. Some estimates put its annual cost as high as $657,000. Each vial of the drug is reported to cost $4,215 each, and in the U.S. alone, the 500 Americans who suffer from Hunter syndrome spent a combined $353 million on Elaprase in 2009.

The incurable condition occurs in about one in 150,000 people, almost always males. The disorder typically appears in childhood and impairs growth and mental development. Afflicted children also have a distinct thickening of facial features. A late-onset version of Hunter syndrome is usually milder but can delay growth and damage joints, vision and hearing. Elaprase is an injected medication that replaces the missing enzyme. Manufactured by Shire, it was approved in 2006 improves patients’ ability to walk.

Naglazyne_Galsulfase_$365,000 a year_Maroteaux-Lamy syndrome_Biomarin

Naglazyme

Naglazyme is right behind Elaprase’s reported $375,000 price tag, coming in at the bargain price of just $365,000. This medication is for a rare connective tissue disorder called Maroteaux-Lamy syndrome, which is caused by a deficient enzyme that breaks down large sugar molecules called glycosaminoglycans. The condition occurs in about one of every 200,000 to 600,000 people. The deficiency causes growth retardation in early childhood in addition to heart disease. Naglazyme is made by Biomarin and was approved in 2005. The administration of the drug improves growth and joint movement, as well as range of motion and pain management.

cinryze_C1 esterase inhibitor_$350,000 a year_hereditary angioedema_Viropharma

Cinryze

Patients with hereditary angioedema (HAE) suffer from severe swelling, often in the face and airways, caused by low levels or improper function of the C1 inhibitor protein. This condition is hereditary, and there’s usually a family history, but often, deaths from hereditary angioedema go undiagnosed and reported as a sudden and premature death of a family member. This makes the condition relatively rare, and the treatment is quite expensive: an estimated $350,000 per year for Cinryze, an injectable man-made protein form of complement C1 esterase inhibitor. Cinryze maker Viropharma has mapped out yearly sales of the drug ranging from $95 million to as much as $350 million.

folotyn_Pralatrexate injection_$320,000 a year_peripheral T-cell lymphoma_Allos Therapeutics

Folotyn

This medication is used to treat an aggressive type of cancer of the lymph system, peripheral T-cell lymphoma, which has spread throughout the body. The lymph system is comprised of white blood cells and T cells which fight viruses. There are many types of lymphoma, but T-cell lymphoma is rare, affecting about one to two people per 100,000.

Folotyn, made by Allos Therapeutics, was approved in 2009 and helps prolong survival. Typically, patients will take the drug for about six weeks, but even in that short amount of time, the bill for this treatment is staggering — around $30,000 per month. It’s given to people who have exhausted all other options for treatment and whose cancer has recurred. The drug, an injection, is thought to kill cancer cells; however studies so far have not shown that it prolongs survival. Still, Folotyn was approved as part of the FDA’s accelerated drug approval process to address the needs of people who have a poor prognosis.

Myozyme

Developed by Genzyme, Myozyme costs up to $100,000 per year for child treatment, and about $300,000 per year for adults. Myozyme was created to treat a rare and often fatal disease, Pompe, which disables the heart and skeletal muscles. Often affecting infants, most of its sufferers die in the first year, and those who do survive typically need assistance like ventilators and wheelchairs. But thanks to Myozyme, some patients can do fairly well with the disease, able to speak, walk, and feed themselves. The drama behind creating such an expensive, yet lifesaving drug, was depicted in the movie Extraordinary Measures, sharing the race against time and profit motives experienced in the drug’s development.

Acth

If you think $30,000 per month is insane, consider this: it’s a bargain compared to the approximate $115,000 per month families pay for ACTH. This drug is used to treat infantile spasms, seizures that often affect infants 4 to 6 months of age. Daily injections of ACTH are given for a period of weeks up to several months. At $23,000 per vial, patients often use 6 to 7 vials per course, and often go through two courses, which adds up to more than $300,000 in prescription drug bills. Unfortunately, ACTH is not FDA-approved to treat infantile spasms, and that means families may have trouble getting their insurance companies to pay for this mind-boggling bill.

Arcalyst

Rare genetic conditions like Familial Cold Auto-inflammatory Syndrome and Muckle-Wells Syndrome are inflammatory disorders that cause the body to develop symptoms without a known cause, including virus and illnesses, and can affect the bones, joints, and major organs, leading to deafness, kidney impairment, and vision loss. These inherited conditions impair the immune systems of sufferers, but with Arcalyst, the symptoms associated with these syndromes can be treated and even prevented. It’s even been found to help prevent gout flares, but all of this helpful treatment comes at a very high cost: a reported $250,000 per year of treatment.

Ceredase/Cerezyme

Patients with Gaucher disease, a condition that causes lumps of fat to build up in various places in the body, including the heart, brain, and spleen, suffer from the disease due to a missing enzyme. With Ceredase, made from human placentas, that enzyme can be replaced. But placentas don’t come cheap: the price of this drug is $150,000 per year. A new version, Cerezyme, came out in 1994, made with genetically engineered hamster cells, and was expected to be cheaper, but unfortunately for Gaucher disease sufferers, the price has actually gone up to $200,000 per year for the average patient. The drug has annual sales of more than a billion dollars.

Fabrazyme

Like so many other terribly expensive drugs on this list, Fabrazyme replaces a necessary enzyme in the human body. Patients with Fabry disease suffer from the lack of or faulty enzyme that is needed to metabolize lipids. Without it, lipids are not effectively broken down, and can build to harmful levels in the nervous system, cardiovascular system, eyes, and kidneys, leading to cloudiness of the cornea, increased heart attack and stroke risk, as well as an enlarged heart and impaired kidneys. It’s not hard to understand why this condition is just downright harmful, and why it’s so important to treat. Using Fabrazyme, patients can make up for their enzyme deficiency, reducing deposits throughout the body. The treatment is reported to cost $200,000 for a year of treatment, that is, if you can get it: in 2009, Fabrazyme maker Genzyme’s plant was shut down due to contamination, and is just now resolving its manufacturing problems.

Aldurazyme

Aldurazyme is used to treat a genetic enzyme condition, a far too common and expensive issue on this list. The condition in this case is Hurler syndrome, a metabolic disorder in which the lack of an enzyme keeps the body from breaking down certain sugars and proteins properly. Like Fabry disease, sugars and proteins not broken down will build up, leading to enlarged organs, breathing issues, decreased physical abilities, and more. With Aldurazyme, breathing and walking ability can be improved, but it does cost a pretty penny: $200,000 per year. The drug is usually given on a weekly basis in a clinic or hospital setting, which may incur additional costs as well.

%d bloggers like this: