Ignyta, Inc.’s (RXDX) lead candidate, entrectinib (RXDX-101), has received orphan drug status as well as rare pediatric disease designation from the FDA for the treatment of neuroblastoma. Entrectinib is currently being evaluated in two phase I/II studies – STARTRK-1 and ALKA-372-001.
We note that the FDA usually grants orphan drug status to novel candidates or biologics being developed as a potential treatment for rare diseases/conditions that affect less than 200,000 people in the U.S. This designation allows the product to enjoy a seven-year period of marketing exclusivity in the U.S., upon approval.
Moreover, Ignyta is now eligible to receive tax credits for research and development costs incurred, seek study design assistance from the FDA and apply for annual grant funding as well as request for the waiving of Prescription Drug User Fee Act filing fees.
Additionally, rare pediatric disease designation for entrectinib provides Ignyta the opportunity to obtain a Pediatric Disease Priority Review Voucher from the FDA after a qualifying new drug application (NDA) or biologics license application (BLA) is approved for the treatment of any rare pediatric disease (that affects less than 200,000 individuals in the U.S. in the age group of 0 year – 18 years). The company may use the voucher to obtain priority review for an NDA or BLA submitted later. As an added advantage, Ignyta might sell or transfer the voucher in the future.
Meanwhile, Ignyta is also seeking orphan drug designation for entrectinib from the FDA in several other indications affecting adult patients. The company has a license agreement with Nerviano Medical Sciences for the development and commercialization of entrectinib.
Ignyta is also working on the development of its oncology pipeline. The company has recently added RXDX-103 and RXDX-104 to its pipeline. It intends to take over the sole responsibility of the development and commercialization of RXDX-104 from Nerviano by year-end.
The receipt of orphan drug status coupled with rare pediatric disease designation for entrectinib is encouraging. We expect investors to focus on development updates on the candidate, going forward.
Ignyta carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the biotech sector are The Medicines Company (MDCO), Regado Biosciences, Inc. (RGDO) and Biogen Idec Inc. (BIIB). While The Medicines Company and Regado carry a Zacks Rank #1 (Strong Buy), Biogen holds a Zacks Rank #2 (Buy).
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