The US Food and Drug Administration (FDA) recently accepted the Biologics License Application (BLA) for Santarus, Inc. and partner, Pharming Group NV’s Ruconest. The companies are looking to get Ruconest approved for the treatment of hereditary angioedema (HAE) patients suffering from acute angioedema attacks.
While the FDA said that it will respond on the BLA by Apr 16, 2014, the agency mentioned that the BLA will be reviewed by its Blood Products Advisory Committee.
A phase III randomized placebo-controlled study was conducted under the FDA’s Special Protocol Assessment program to evaluate Ruconest’s safety and efficacy. Santarus also conducted two additional randomized placebo-controlled and open-label treatment studies on Ruconest.
Ruconest is already approved in Europe for the same indication. We note that it enjoys orphan drug status in the US for treating acute attacks of HAE as well as for the prophylactic treatment of HAE. Once approved, Ruconest could have 12 years of data exclusivity.
Santarus has a licensing and supply agreement with Pharming for Ruconest. Santarus gained some non-exclusive rights for development and manufacturing, along with some exclusive rights for commercializing Ruconest in the US, Canada and Mexico for treating HAE and other future indications. The company intends to initiate a proof-of-concept study on Ruconest later this year for the treatment of acute pancreatitis.
As per the agreement, Santarus made an upfront payment of $15 million as well as a milestone payment of $10 million on the successful achievement of the primary endpoint of the phase III study.
According to the US Hereditary Angioedema Association, about one out of 10,000 to one out of 50,000 individuals suffer from HAE. Currently approved therapies include ViroPharma Inc.’s Cinryze and Shire plc’s (SHPG) Firazyr.
Santarus currently carries a Zacks Rank # 1 (Strong Buy). The company received a major boost earlier this year with the US launch of Uceris (the induction of remission in patients suffering from mild-to-moderate ulcerative colitis). Uceris is off to a strong start and should continue performing well. We expect investor focus to remain on the Uceris launch, the Zegerid re-launch and the outcome of the FDA advisory panel meeting for Ruconest.
At present, companies like Jazz Pharmaceuticals (JAZZ - Analyst Report), also carry a comparable rank.