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Home » FDA 2014 » FDA Approves Vimizim to Treat Mucopolysaccharidosis Type IVA

FDA Approves Vimizim to Treat Mucopolysaccharidosis Type IVA

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STRUCTURAL FORMULA
Monomer
APQPPNILLL LMDDMGWGDL GVYGEPSRET PNLDRMAAEG LLFPNFYSAN 50
PLCSPSRAAL LTGRLPIRNG FYTTNAHARN AYTPQEIVGG IPDSEQLLPE 100
LLKKAGYVSK IVGKWHLGHR PQFHPLKHGF DEWFGSPNCH FGPYDNKARP 150
NIPVYRDWEM VGRYYEEFPI NLKTGEANLT QIYLQEALDF IKRQARHHPF 200
FLYWAVDATH APVYASKPFL GTSQRGRYGD AVREIDDSIG KILELLQDLH 250
VADNTFVFFT SDNGAALISA PEQGGSNGPF LCGKQTTFEG GMREPALAWW 300
PGHVTAGQVS HQLGSIMDLF TTSLALAGLT PPSDRAIDGL NLLPTLLQGR 350
LMDRPIFYYR GDTLMAATLG QHKAHFWTWT NSWENFRQGI DFCPGQNVSG 400
VTTHNLEDHT KLPLIFHLGR DPGERFPLSF ASAEYQEALS RITSVVQQHQ 450
EALVPAQPQL NVCNWAVMNW APPGCEKLGK CLTPPESIPK KCLWSH 496
Disulfide bridges
139-139′ 282-393 282′-393′ 463-492 463′-492′ 475-481 475′-481′
Modified residues
C
53 , 53′
3-oxoAla
O
CO2H
H NH2

Glycosylation sites (N)
Asn-178 Asn-178′ Asn-397 Asn-397′

Vimizim (elosufase alfa)

Elosulfase alfa nonproprietary drug name  GET STRUCTURE

MOLECULAR FORMULA C5020H7588N1364O1418S34

MOLECULAR WEIGHT 110.8 kDa (peptide)

SPONSOR BioMarin Pharmaceutical Inc.

CODE DESIGNATION BMN 110, rhGALNS

CAS REGISTRY NUMBER 9025-60-9

THERAPEUTIC CLAIM Treatment of Morquio Syndrome

CHEMICAL NAMES

1. Sulfatase, chondroitin

2. Human N-acetylgalactosamine-6-sulfatase (chondroitinsulfatase, galactose-6-sulfate
sulfatase, EC=3.1.6.4) dimer (139-139′)-disulfide glycosylated (produced by CHO cells)

Company: BioMarin Pharmaceutical Inc.
Date of FDA Approval: February 14, 2014
Treatment for: Mucopolysaccharidosis Type IVA

  • BMN 110
  • Chondroitin 6-sulfatase
  • Chondroitin sulfatase
  • Chondroitin sulfate sulfatase
  • Chondroitinase
  • Chondrosulfatase
  • E.C. 3.1.6.4
  • Elosulfase alfa
  • rhGALNS
  • UNII-ODJ69JZG85
  • Vimizim

CLINICAL….http://clinicaltrials.gov/search/intervention=Elosulfase%20alfa%20OR%20bmn%20110

Vimizim (elosufase alfa) is an enzyme replacement therapy for patients with Mucopolysaccharidosis Type IVA (Morquio A syndrome).

  • FDA Advisory Committee Recommends Approval for BioMarin’s Vimizim for the Treatment of Patients With Morquio A Syndrome – November 20, 2013

Feb 16, 2014 Approval FDA Approves Vimizim to Treat Mucopolysaccharidosis Type IVA

The U.S. Food and Drug Administration today approved Vimizim (elosulfase alfa), the first FDA-approved treatment for Mucopolysaccharidosis Type IVA (Morquio A syndrome). Morquio A syndrome is a rare, autosomal recessive lysosomal storage disease caused by a deficiency in N-acetylgalactosamine-6-sulfate sulfatase (GALNS). Vimizim is intended to replace the missing GALNS enzyme involved in an important metabolic pathway. Absence of this enzyme leads to problems with bone development, growth and mobility. There are approximately 800 patients with Morquio A syndrome in the United States.

Vimizim was granted priority review. An FDA priority review provides for an expedited review of drugs for serious diseases or conditions that may offer major advances in treatment. Vimizim is also the first drug to receive the Rare Pediatric Disease Priority Review Voucher – a provision that aims to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

“This approval and rare pediatric disease priority review voucher underscores the agency’s commitment to making treatments available to patients with rare diseases,” said Andrew E. Mulberg, M.D., deputy director, Division of Gastroenterology and Inborn Errors Products in the FDA’s Center for Drug Evaluation and Research (CDER). “Prior to today’s approval, patients with this rare disease have had no approved drug treatment options.”

The safety and effectiveness of Vimizim were established in a clinical trial involving 176 participants with Morquio A syndrome, ranging in age from 5 to 57 years. Participants treated with Vimizim showed greater improvement in a 6-minute walk test than participants treated with placebo. On average, patients treated with Vimizim in the trial walked 22.5 meters farther in 6 minutes compared to the patients who received placebo.

The most common side effects in patients treated with Vimizim during clinical trials included fever, vomiting, headache, nausea, abdominal pain, chills and fatigue. The safety and effectiveness of Vimizim have not been established in pediatric patients less than 5 years of age. Vimizim is being approved with a boxed warning to include the risk of anaphylaxis. During clinical trials, life-threatening anaphylactic reactions occurred in some patients during Vimizim infusions.

Vimizim is marketed by Novato, Calif.-based BioMarin Pharmaceutical Inc.

Elosulfase alfa (GALNS), a proposed treatment for Morqio A syndrome. Morquio A syndrome is an inherited, autosomal recessive disease caused by a deficiency of a particular lysosomal enzyme, N- acetylgalactosamine- 6 sulfatase. BioMarin’s experimental drug for Morquio A syndrome is an enzyme replacement of elosulfase alfa (called BMN 110), which is designed to clear keratan sulfate from the lysosome. BMN 110 is being studied to determine if it is safe, if it will slow the progression of the disease and if it will improve some of the symptoms.

BioMarin started BMN 110 clinical studies in humans in 2009 to evaluate safety and efficacy. In a phase III Multicenter, Multinational, Extension Studythe Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) MOR-005 was evaluated. Participants will receive 2 mg/kg weekly or every other weekly dosing of study drug via infusion until the MOR- 004 study is unblinded and the optimal dose is selected. All subjects will then be treated with the optimal dose for up to approximately 5 years or until the drug is approved.

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DR ANTHONY CRASTO

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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 29 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 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 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 29 year tenure till date Aug 2016, Around 30 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, 25 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 13 lakh plus views on New Drug Approvals Blog in 212 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

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