Editas, Neurocrine Snag Orphan Drug Designations as Congressional Debate Continues

As the fairness of orphan drug exclusivities is debated in Congress, the U.S. Food and Drug Administration granted Orphan Drug designations to Editas Medicine and Neurocrine Biosciences this week.

The FDA awarded the designation to Editas for its experimental gene therapy EDIT-301, under development for the treatment of beta thalassemia. EDIT-301 was previously awarded Rare Pediatric Disease designation for the treatment of beta thalassemia and sickle cell disease. The gene therapy is currently being assessed in the RUBY trial for patients with severe sickle cell disease and Editas intends to begin a Phase I/II study of EDIT-301 in patients with transfusion-dependent beta thalassemia later this year.

James C. Mullen, chairman, president and chief executive officer of Editas, said receiving Orphan Drug designation for EDIT-301 in beta thalassemia highlights the need for new treatment options for these patients. Mullen said the company hopes to dose the first beta thalassemia patient later this year.

San Diego-based Neurocrine also picked up the designation for its experimental Huntington’s disease treatment valbenazine, a selective vesicular monoamine transporter 2 (VMAT2) inhibitor. Neurocrine noted that the treatment of chorea associated with Huntington’s disease is within the scope of this Orphan Drug designation. Late last year, Neurocrine reported positive top-line data from the KINECT-HD study evaluating the efficacy, safety and tolerability of valbenazine.

We are in the process of completing data analysis from the KINECT-HD and the ongoing KINECT-HD2 studies, which will form the basis of our supplemental new drug application (sNDA) for submission to the FDA later this year,” Neurocrine CEO Kevin Gorman said in a statement.

Neurocrine is enrolling patients in its KINECT-HD2 open-label study assessing the long-term safety and tolerability of valbenazine for the treatment of chorea in Huntington’s disease.

An Orphan Drug designation confers the orphan status to drugs or biologics developed for the treatment of diseases that affect fewer than 200,000 people in the United States. Within the designation is a seven-year marketing exclusivity upon approval from the FDA. It’s that exclusivity that Congress is investigating as a potential monopoly. A bill that has passed the U.S. House of Representatives, will limit which orphan drugs may be granted that seven-year market exclusivity period by the FDA.

The legislation dubbed the Fairness in Orphan Drug Exclusivity Act, indicates that the designation and exclusivity will be designated only if a drug developer can prove that it will not likely recover costs of developing and distributing the medication within the U.S. after the first 12 years of commercial availability. The requirement shall also apply retroactively to an orphan drug that was granted such an exclusivity period before this bill’s enactment.

“When deciding whether an orphan drug meets this requirement, the FDA shall consider the sales of all drugs from the sponsor that are covered by the same orphan drug designation,” the legislation reads.

While Orphan Drug designation is discussed in the halls of Congress, an international consortium has formed the World Orphan Drug Alliance (WODA), which was formed to “provide comprehensive support to pharmaceutical and biotech companies with rare diseases, oncology and highly specialized therapeutics portfolio, starting from named patient programs through to full commercialization.”

WODA covers 68 countries across Asia, Africa, Latin America, parts of Europe and Switzerland. The organization aims to provide all necessary services to ensure orphan drugs reach patients in need throughout these areas.

“WODA focuses on the markets that are complex and often overlooked by big pharmaceutical and biotech companies. However, WODA territories provide both a pool of patients in need and potential funding opportunities for rare and specialized diseases. There are potentially more than 64 million rare disease patients in the WODA regions,” Martina Perharič, chief executive officer of Slovenia-based Medis, a medical marketing company that is part of WODA said in a statement.