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7 Not To Miss February FDA Regulatory Events And Meetings For Orphan Drugs

February 4, 2013

I – Ampligen (Rintatolimod)

The FDA was expected to make a decision on Ampligen for CFS by the PDUFA date of February 2, 2013. The date came and went with no announcement. Hopefully early in the week there will be a decision.

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 isn’t required to follow the advice of the Advisory Committee, but usually does. Reference the Blog Post for the complete story.

II – 5th International Meeting on Pulmonary Rare Diseases & Orphan Drugs

Milan, Italy, Feb 8-9, 2013

III – Pomalyst (Pomalidomide)

Investors will be closely watching the February 10, 2013 scheduled FDA PDUFA date for Celgene’s orphan drug Pomalyst (Pomalidomide) for the treatment of Relapsed and Refractory Multiple Myeloma (RRMM). Both the FDA and the European Medicines Agency (EMA) are reviewing applications for the approval of Pomalyst in combination with Dexamethasone for the treatment of RRMM patients who have received at least 2 prior therapies. Reference Blog Post for the complete story.

IV – BIO CEO & Investor Conference

The BIO CEO & Investor Conference takes place in New York February 11-12. The event features presentations by dozens of pharmaceuticals and biotechs. CEOs from several companies will be in the spotlight during public interviews with industry analysts.

V – Lysosomal Disease Network (LDN) 9th Annual World Symposium

Orlando, Florida, USA, Feb 12 – 15, 2013

Presentations for Gaucher Disease:

Genzyme’s Eliglustat Tartrate Presentations : Friday, February 15, 2013 (Session 5 – Clinical Research)

8:30 AM M. Judith Peterschmitt – Biomarker Responses to Eliglustat, an Investigational Oral Substrate Reduction Therapy (SRT) for Gaucher Disease Type 1

11:15 AM Pramod Mistry – Phase III (ENGAGE), Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Investigate The Efficacy & Safety of Eliglustat in Patients with Gaucher Disease: Results After 9 Months of Treatment.

Pfizer & Protalix BioTherapeutics’ Elelyso Presentations : Friday, February 15, 2013 (Session 5 – Clinical Research)

11:00 AM  – Gregory Pastores  – Plant Cell-Expressed Recombinant Glucocerebrosidase: Taliglucerase Alfa as Therapy for Gaucher Disease in Adults Patients Previously Treated with Imiglucerase: 24-Month Results.

Additionally, Professor Ari Zimran, M.D., Director of the Gaucher Clinic, Israel, will present 2 Posters :

  1. “Long-Term Safety and Efficacy Data of Taliglucerase Alfa, a Plant Cell–Expressed Recombinant Glucocerebrosidase, in the Treatment of Naïve Gaucher Disease Patients: 36-Month Results”
  2. “A Multicenter, Double-Blind, Randomized Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Patients with Gaucher Disease”.

Shire’s Vpriv Presentation : Friday, February 15, 2013 (Session 5 – Clinical Research)

10:45 AM Ari Zimran  – Bone Mineral Density Change in Type 1 Gaucher Disease Adults Given Velaglucerase Alfa for 2 Years.

VI – 6th Annual World Rare Disease Day 2013

February 28, 2013

VII – Stivarga (Regorafenib)

The FDA PDUFA date for Bayer and Onyx Pharmaceuticals’ Stivarga (Regorafenib), for the treatment of Gastrointestinal Stromal Tumors (GIST), is February 28, 2013.

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

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