Dive Brief:
-
Biogen, buoyed by encouraging data on its spinal muscular atrophy (SMA) drug, said the "first key element" ahead of a U.S. launch will be engaging the community of "highly informed," "engaged and motivated" patients and their families.
-
Increased demand from such a galvanized patient community will require adjustments to infrastructure and logistics, Biogen said Monday at a Credit Suisse healthcare conference. This may necessitate multidisciplinary teams, including anesthesiologists, to administer nusinersen to infants or babies with SMA.
-
Only 20 of approximately 40 to 50 SMA centers in the U.S. participated in clinical trials for nusinersen, but even those centers likely will need to adjust capacity based on an increased inflow of patients, Chief Commercial Officer Michel Vounatsos said.
Dive Insight:
While Biogen's experimental SMA therapy remains under regulatory review, the company's execs said nusinersen's path toward approval is “progressing well,” and the company expects an earlier-than-expected approval.
"In terms of launch readiness, we are accelerating our activities for a potential launch towards the end of the year or early 2017," said Vounatsos. Those activities will be focused at engaging patients and families, as well as building out the infrastructure and expertise needed to administer the intrathecal injection for nusinersen.
Results from the most recent Phase 3 trial, which studied nusinersen in later-onset SMA, showed the drug helped improve motor function compared to placebo. This complements earlier positive results from another Phase 3 study in infantile-onset SMA.
Taken together, analysts see the data as supportive of a broad label for nusinersen, which would boost a potential launch of the drug.
According to figures cited by the SMA Foundation, approximately 1 in 6,0000 to 1 in 10,000 children are born with SMA. While incidence of the more severe, early-onset form of SMA (type 1) is much higher than the later-onset versions of the condition (types 2 and 3), more than half of living SMA patients have type 2 SMA due to the high fatality rates in type 1 SMA.
Apart from the U.S., Biogen is moving forward in other regulatory areas as well. Asked about Biogen's strategy in Japan, Vounatsos said the company is working with Japan's Pharmaceuticals and Medical Devices Agency "so that we can be ready for an as early as possible filing in Japan."