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Orphan Drugs And Rare Diseases: Global News (07.02.13)

July 2, 2013

Recent orphan drug and rare disease global developments are in the news. Here is a list and description of recent developments:

I – Europe

The European Union Committee of Experts on Rare Diseases (EUCERD) adopts a set of recommendations, at its June 2013 meeting, for patient registration and data collection for rare disease registries. A document of the recommendations are available.

In June 2013, Raptor Pharmaceutical, a California company, announces that the European Committee for Medicinal Products for Human Use (CHMP) adopts a positive opinion recommending marketing authorization for orphan drug Procysbi (Cysteamine) for the treatment of proven Nephropathic Cystinosis. The European Commission (EC) must ratify CHMP’s positive opinion in order to grant marketing authorization for Procysbi. This ratification by the EC will cover 27 EU countries. The decision is expected from the EC in the second half of 2013. The recent history of Procysbi’s regulatory events:

•   Receives FDA approval in April 2013
•   Commercially available in US in June 2013
•   Receives FDA Orphan Drug Exclusivity in June 2013.

“Nephropathic cystinosis comprises 95% of cases of cystinosis, a rare, life-threatening metabolic lysosomal storage disorder that causes toxic accumulation of cystine in all cells, tissues, and organs in the body. Elevated cystine leads to progressive, irreversible tissue damage and multi-organ failure, including kidney failure, blindness, muscle wasting and premature death. Nephropathic cystinosis is usually diagnosed in infancy and requires lifelong therapy. Left untreated, the disease is usually fatal by the end of the first decade of life. There are an estimated 500 patients living in the U.S. with cystinosis, and 2,000 worldwide.”

II – Brazil

Genzyme announces July 1, 2013, the 1st International Gaucher Conference on Bone Disease, July 5 – 6, 2013, Rio de Janeiro, Brazil. Over 100 global researchers and physicians will gather with the goal of “improving clinical care and shaping the future direction of research specific to bone disease” in patients with Gaucher Disease. Some of the topics to be covered are:

•   Management of bone disease in children & adults
•   Pathophysiology of bone disease in Gaucher Disease
•   Biomarkers & bone turnover markers
•   Radiological assessment of bone disease.

Orphan drug access in Brazil receives a boost from May 2013 legislation introduced in the National Congress. The bill combines three earlier measures with the purpose of establishing national coverage for drugs for rare diseases.

III – Taiwan

In June 2013, a research team from Academia Sinica and the National Taiwan University (NTU), produces the “world’s first induced pluripotent stem cells (iPSC) for Pompe Disease.” Pompe Disease is a rare genetic disease caused by acid maltase deficiency. The incidence rate in Taiwan is slightly higher than in other countries.

“The council-funded research team said it has successfully generated Pompe Disease-iPSC from the fibroblasts — a type of cell — of patients, by applying a temporary genetic rescue method, becoming the first Pompe Disease-iPSC recognized by the international medical society.” 

IV – United States

According to a July 1, 2013 Press Release from Thomson Reuters and the National Venture Capital Association (NVCA), the second quarter 2013 saw “the highest number of biotechnology venture-backed IPOs since the third quarter of 2000”. The following statistics are reported:

•   13 of the 21 offerings during the 2nd Q2013 are Life Sciences IPOs representing 62% of total issues for the quarter
•   11 of the 13 Life Sciences IPOs during the 2nd Q2013 are biotechnology offerings – strongest Quarter for listings in the sector since the 3rd Q2000
•   20 of the 2nd Q2013 IPOs are from US-based companies.

Several of the biotechnology companies are in the orphan drug and rare disease space:

•   Prosensa raises $78 million (6 million shares offered at $13/share) on the NASDAQ stock exchange on June 28, 2013, to support its products for the rare disease, Duchenne Muscular Dystrophy (DMD), and other programs
•   Bluebird bio, a biotechnology company developing gene therapies for severe genetic and rare diseases, announces on June 24, 2013, the closing of its IPO of 6,832,352 shares of common stock at an initial public offering price of $17/share and raising approximately $116.15 million
•   PTC Therapeutics closes on June 25, 2013 its IPO of 9,627,800 shares of common stock at a public offering price of $15/share.

The FDA in June 2013 issues a draft guidance outlining both the “qualifying criteria and benefits for the agency’s expedited programs for serious or life-threatening conditions.” The four expedited programs that are reviewed are:

•   Fast Track
•   Breakthrough Therapy
•   Accelerated Approval
•   Priority Review.

Comments on the draft guidance are due August 26, 2013. Further discussion will be provided by the Blog this week.

Please Note: “Globe” by Augiasstallputzer at Wikimedia Commons [Public domain] | Wikimedia Commons.

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

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