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3 Not To Miss FDA Regulatory Events This Week For Orphan Drugs

January 28, 2013

I – Kynamro (Mipomersen)

Investors are keeping a close eye on the FDA’s ruling on Isis Pharmaceuticals and Genzyme’s orphan drug Kynamro, which is expected on Tuesday, January 29, 2013. Kynamro is for the treatment of Homozygous Familial Hypercholesterolemia (HoFH).  An Orphan Druganaut Blog Post last week reviewed in detail the regulatory timeline and the comparison of Kynamro verses Aegerion Pharmaceuticals’ orphan drug Juxtapid (Lomitapid) for the same indication.

Both competitive drugs for HoFH are proven effective. It is the question of safety which will determine what drug becomes the winner in the market.  While the FDA’s Endocrinologic & Metabolic Drug Advisory Committee unanimously recommended Juxtapid, the same Committee approved Kynamro by a 9-6 vote. The Committee was concerned with the excess fat buildup in the liver and abnormal neoplasm growths. Juxtapid was approved before its PDUFA date. Also, Kynamro is issued a negative opinion by the European Medicines Agency’s (EMA) Medicinal Products for Human Use (CHMP) in December 2012 because of the drug’s safety.

 PharmaLive.com recently interviewed Paula Soteropoulos, Genzyme’s Vice President and General Manager of cardiovascular business, about Kynamro :

–  Kynamro will launch with a very targeted medical team and sales force

–  Patient support system that will help with care, insurance, and co-pays

–  Working with EMA’s CHMP on a re-examination request, with an expected opinion in the 2nd Quarter 2013.

II – Bronchitol (Mannitol)

This Wednesday,January 30, 2013, the FDA Pulmonary-Allergy Drugs Advisory Committee Meeting will “discuss the New Drug Application (NDA) 202049, for mannitol inhalation powder (proposed trade name Bronchitol) for oral inhalation sponsored by Pharmaxis, for the proposed indication of the management of Cystic Fibrosis (CF) in patients aged 6 years and older to improve pulmonary function”.

Pharmaxis is an Australian pharmaceutical company specializing in undertreated respiratory diseases. Bronchitol has Orphan Drug Designation in the US, Europe, and Australia. It is approved for marketing in Australia and throughout the European Union.

III – Ampligen (Rintatolimod)

The FDA Arthritis Advisory Committee Meeting on December 20, 2012, decides in an 8-to-5 vote that Hemispherx BioPharma’s orphan drug Ampligen (Rintatolimod) for the treatment of Chronic Fatigue Syndrome (CFS) isn’t ready for approval. Per the Committee, this vote was a result from the company not providing sufficient data.

The FDA expects to make a decision on Ampligen for CFS by the PDUFA date of February 2, 2013. The FDA isn’t required to follow the advice of the Advisory Committee, but usually does.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved

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