BioCryst Pharmaceuticals, Inc. (BCRX) announced that the company has received a positive opinion regarding its application for orphan drug status for BCX4161 from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency. The company is developing BCX4161 for the treatment of patients suffering from hereditary angioedema (HAE).
Currently, BCX4161 is in a double-blind, randomized, placebo-controlled study (OPuS-2) evaluating the safety and efficacy of the candidate in treating HAE patients for 12 weeks. Results from the OPuS-2 study should be out by the end of 2015.
Although it is not compulsory for the European Commission to follow the COMP’s opinion, it usually considers the same while making a decision.
In the EU, orphan drug status is granted to a therapy that treats or aims to treat a life-threatening or a chronically debilitating condition affecting ≤5 patients in 10,000 people and for which a satisfactory treatment is not yet available or a new treatment is expected to provide significant benefit to the patients. While orphan drug designation does not change the standard regulatory requirements for attaining marketing approval for a candidate, companies may seek protocol assistance from the EMA during the development phase and receive regulatory and financial incentives for developing and marketing such therapies. This designation also provides a 10-year period of marketing exclusivity in the EU after the product is approved.
According to the press release issued by BioCryst, HAE is a severely debilitating and potentially fatal genetic condition affecting about 1 in 50,000 people. Orphan drug designation for BCX4161 should accelerate the development process for the candidate.
We note that BCX4161 also enjoys orphan drug designation in the U.S. for the prevention of acute attacks of angioedema in patients suffering from HAE.
Notably, Shire’s (SHPG) Firazyr is already approved for the treatment of acute HAE attacks in patients aged 18 years and above.
BioCryst carries a Zacks Rank #1 (Strong Buy). Other well-ranked stocks in the health care sector are Endo International plc (ENDP) and Auxilium Pharmaceuticals Inc. (AUXL). While Biodel and Endo carry a Zacks Rank #1, Auxilium holds a Zacks Rank #2 (Buy). Shire also carries a Zacks Rank #2.
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