On Tuesday, the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) for Biogen Inc.’s (NASDAQ: BIIB) supplemental New Drug Application (sNDA) for the high-dose regimen of nusinersen for the treatment of spinal muscular atrophy (SMA).
The FDA letter requested an update to the technical information be included in the Chemistry Manufacturing and Controls (CMC) module of the sNDA.
The letter did not cite any deficiencies in the clinical data of the high-dose regimen. The FDA provided options for resolution, and Biogen is planning to resubmit the application promptly based on readily available information.
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“While this outcome was unexpected, we remain committed to bringing the high-dose regimen to people living with SMA,” said Priya Singhal, Head of Development at Biogen. “We are working diligently to provide the necessary information to the FDA.”
The high-dose regimen of SPINRAZA (nusinersen) was recently approved in Japan and is actively under review by the European Medicines Agency (EMA) and other global regulators.
On Wednesday, William Blair said, “The CRL for the high dose of Spinraza comes as a surprise and will be viewed as an incremental negative for Biogen, causing minimal stock volatility. However, since the CRL was related only to CMC deficiencies, we see a relatively straightforward path to resolution here, and continue to believe the high-dose regimen will garner eventual approval.”
Analyst Myles R. Minter added that investors will watch pricing closely, as DEVOTE Part B suggests the 28 mg Spinraza dose may deliver better event-free survival than the standard 12 mg regimen.
Most recently, Biogen agreed to acquire Massachusetts-based Alcyone Therapeutics for $85 million.
Alcyone will also receive certain milestones payable related to the development and regulatory approval of ThecaFlex DRx with nusinersen and additional pipeline products, securing all rights to ThecaFlex DRx.
The ThecaFlex DRx System is initially being evaluated with Spinraza (nusinersen) in patients with spinal muscular atrophy, which will inform pathways for Biogen's broader portfolio of investigational therapies.
BIIB Price Action: Biogen shares were up 0.24% at $138.00 at the time of publication on Thursday. The stock is trading within its 52-week range of $110.03 to $199.99, according to Benzinga Pro data.
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