Brief

Biogen, Ionis gifted with approval for SMA drug

Dive Brief:

  • After the market closed on Dec. 23, Biogen and Ionis Pharmaceuticals got an early Christmas present from the Food and Drug Administration with the early approval of their spinal muscular atrophy drug Spinraza (nusinersen).
  • The antisense drug had a user fee goal date of May 26, 2017 and is now the first approved treatment for the rare genetic disorder.
  • Spinraza garnered a broad label from the FDA and was approved for all subtypes of the disease, as well as both pediatric and adult patients based on the ENDEAR study. 

Dive Insight:

​Biogen and Ionis could launch Spinraza before the end of 2016 or in early 2017, and analysts believe the drug has the potential to add $1.7 billion to Biogen's revenues by 2025.

The approval was a major win for the company, which has been struggling to prove to investors that its pipeline isn't too risky. It should also give a lift to outgoing CEO George Scangos' successor — Biogen announced that Chief Commercial Officer Michel Vounatsos will take over as CEO as of Jan. 6.

For Ionis, which linked up with Biogen in 2012 when it was still known as Isis Pharmaceuticals, will get a $150 million milestone payment for the approval. Under the original terms of the deal, Biogen paid $29 million upfront and $45 million in clinical milestone payments.

As part of the approval, Biogen also gains a priority review voucher. The coupon could help speed the regulatory process of a drug or can be sold to other companies for that purpose. These vouchers have been going for $250 million to $300 million.

Roche-backed AveXis is also developing an SMA drug and analysts believe there could be room for improvement, despite strong data from Spinraza. AveXis' drug is expected to enter pivotal trials in the first half of 2017.

Spinraza will be given in four loading doses: the first three doses delivered 14 days apart with the last dose 30 days after the third, followed by maintenance doses every four months.

Data from the ENDEAR trial showed that 23% of infants in the Spinraza arm died, compared to 43% of those in the placebo group.

"Our feedback from parents and physicians indicates that despite the approval of Spinraza there remains interest in enrolling [patients] into a clinical trial testing AVXS-101. The ease of dosing, and one time dose are key advantages with AVXS-101," wrote Jefferies analyst Biren Amen in a recent note to investors. 

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Filed Under: Regulatory / Compliance