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Home » FDA 2014 » FDA approves new drug Cerdelga (eliglustat) to treat a form of Gaucher disease

FDA approves new drug Cerdelga (eliglustat) to treat a form of Gaucher disease

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August 19, 2014

Release

The U.S. Food and Drug Administration today approved Cerdelga (eliglustat) for the long-term treatment of adult patients with the Type 1 form of Gaucher disease, a rare genetic disorder.
Gaucher disease occurs in people who do not produce enough of an enzyme called glucocerebrosidase. The enzyme deficiency causes fatty materials to collect in the spleen, liver and bone marrow. The major signs of Gaucher disease include liver and spleen enlargement, low red blood cell counts (anemia), low blood platelet counts and bone problems.
Cerdelga is a hard gelatin capsule containing eliglustat that is taken orally. In patients with Gaucher disease Type 1, the drug slows down the production of the fatty materials by inhibiting the metabolic process that forms them. Type 1 Gaucher disease is estimated to affect about 6,000 people in the United States.
“Today’s approval offers another important treatment option for patients with Type 1 Gaucher disease,” said Amy G. Egan, M.D., M.P.H., deputy director of the Office of Drug Evaluation III in FDA’s Center for Drug Evaluation and Research. “In addition, Cerdelga received orphan drug designation from the FDA, reflecting the agency’s focus and commitment to the development of treatments for rare diseases.”
The safety and effectiveness of Cerdelga were evaluated in two clinical trials with 199 participants with Type 1 Gaucher disease.
In one randomized, double-blind, placebo-controlled, multicenter clinical trial the safety and effectiveness of Cerdelga were evaluated in 40 participants with Type 1 Gaucher’s disease who had not previously received enzyme replacement therapy. Subjects received the drug at a starting dose of 42 mg two times a day, with most receiving a dose of 84 mg two times a day after four weeks. Study participants continued the drug for nine months.
Compared to placebo, treatment with Cerdelga resulted in a greater reduction in spleen volume from baseline to the end of the study (by the 39th week), the trial’s primary endpoint. Cerdelga also resulted in greater improvement in liver volume, blood platelet count, and red blood cell (hemoglobin) level, compared to placebo.
The other trial sought to determine the safety and effectiveness of Cerdelga compared to enzyme replacement therapy in 159 participants with Type 1 Gaucher disease previously treated and stabilized on enzyme replacement therapy. Subjects in the trial received either the enzyme replacement therapy drug imiglucerase or Cerdelga. The trial demonstrated that treatment with Cerdelga resulted in similar stabilization of hemoglobin level, platelet count and spleen and liver volume as imiglucerase.
The most commonly observed side effects in the Cerdelga clinical trials were fatigue, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain.
Cerdelga is manufactured by Cambridge, Massachusetts-based Genzyme.

 

read synthesis at
https://newdrugapprovals.org/2013/12/14/the-us-food-and-drug-administration-fda-has-granted-a-six-month-priority-review-designation-to-genzymes-new-drug-application-nda-for-cerdelga-eliglustat/ – See more at: http://worlddrugtracker.blogspot.in/#sthash.tJzVgHVT.dpuf

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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 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

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