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Orphan Drugs: Making Rare Diseases Rarer

February 25, 2013
A panel at the BIO CEO & Investor Conference provided an analysis of the thriving orphan drug market opportunity featuring industry, clinical and regulatory experts specializing in rare diseases.

The signing of FDASIA by President Obama in July reinforces the country’s commitment to fostering innovation for rare diseases. With a record 460 medicines in late-stage clinical trials, biopharmaceutical companies have embraced rare disease medicine and are rapidly developing the pipeline with hopes of achieving the ever elusive blockbuster drug.

Some companies have even taken a step beyond and focused primarily on ultra rare disease, potentially providing an even more compelling opportunity.

Alvin V. Shih, MD, Chief Operating Officer of the Pfizer Rare Disease Research Unit kicked off the session by noting that there were 39 new approvals in 2012, which is a sign that "the FDA is rewarding companies that innovate."

John F. Crowley, Chairman & CEO of Amicus Therapeutics added that the FDA has made great strides in the past two years and that we’re now moving in the right direction in terms of the regulatory environment, and that we are now on the ‘cusp of some positive developments in the future.’

Marc Beer, Chief Executive Officer of Aegerion Pharmaceuticals shared his company’s experience on the recent approval of Juxtapid to treat a rare cholesterol disease. “The FDA took the time to understand the morbidity and mortality implications and, therefore, understand the drug at a deeper level; it was a two-way dialogue with the agency from pre-filing all the way through to approval.”

Hans GCP Schikan, PharmD, Chief Executive Officer of Prosensa commented that the involvement of patient groups has created a ‘seismic shift’ in the FDA’s approach to drug reviews and approvals.

Mary Dunkle, Vice President of Communications at the National Organization for Rare Disorders emphasized that we need to consider the cost of failing to treat orphan diseases.

Ritu S. Baral, Principal, Sr. Analyst – Biotechnology Equity Research at Canaccord Genuity moderated the session.