FDA Approves First-Ever Treatment for Spinal Muscular Atrophy; Ionis Pharma, Biogen Seal Victory

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December 27, 2016
By Alex Keown, BioSpace.com Breaking News Staff

CAMBRIDGE, Mass. – Biogen received an early Christmas present from the U.S. Food and Drug Administration with the approval of its spinal muscular atrophy treatment, Spinraza. This is the first drug of its type approved for the rare disease.

The FDA handed down its decision Dec. 23. Biogen’s Spinraza (nusinersen) is the only treatment in the United States approved for SMA, a leading genetic cause of death in infants and toddlers that is marked by progressive, debilitating muscle weakness. Spinraza is an antisense oligonucleotide (ASO) that is designed to alter the splicing of SMN2, a gene that is nearly identical to SMN1. Biogen co-developed Spinraza with Ionis Pharmaceuticals . Spinraza has the potential for the broadest possible label expansion for SMA Types 1, 2, and 3. The drug has been in clinical development since 2011.

SMA is a disorder characterized by loss of motor neurons in the spinal cord and lower brain stem. This results in severe and progressive muscular atrophy and weakness. Over time, patients with the most severe form of SMA can become paralyzed and have difficulty in breathing and swallowing. Patients with SMA do not produce enough survival motor neuron (SMN) protein, which is caused by a defect in the SMN1 gene.

Shares of Biogen jumped on Dec, 23 following the FDA’s announcement and have continued to climb since. This morning, shares of Biogen are trading at $294.30. Ionis stock also saw a jump following the ruling. Shares of Ionis are trading at $56.25 this morning.

As a result of the FDA approval of Spinraza, Ionis is set to receive a milestone payment of $60 million. Ionis is also eligible to receive tiered royalties on any potential sales of Spinraza up to a percentage in the mid-teens, Biogen said. No price tag for Spinraza has yet been disclosed, but it is expected to come with a hefty cost, with some predicting the drug could command a price tag of up to $250,000. Analysts predict the drug could generate up to $3 billion in annual sales by 2026.

The FDA approval of Spinraza was based on positive results from multiple clinical studies in more than 170 patients.

Biogen and Ionis are moving quickly to make Spinraza available for patients. On Dec. 23, the company said it will make the therapy available for shipment to healthcare providers in the United States in approximately one week–meeting the timeline the company had initially set for early January 2017. Biogen anticipates there may be variation in time to treatment as institutions and treatment centers learn about Spinraza, the company said in its statement.

“Spinraza offers new hope for the SMA community and exemplifies our mission of applying cutting-edge science to make a meaningful difference in the lives of patients with devastating, life-altering diseases,” George Scangos, Biogen’s chief executive officer, said in a statement. “We are humbled and grateful for the commitment of the patients and families who participated in the Spinraza clinical trial program, the tireless efforts of our investigators, and the urgency demonstrated by the FDA in rapidly reviewing and approving this treatment. We also want to acknowledge the important work of our colleagues at Ionis, who initiated this program.”

In October, the European Medicines Agency approved Biogen’s Marketing Authorization Application for Spinraza. The EMA’s Committee for Medicinal Products for Human Use granted Accelerated Assessment status. In addition, Biogen has submitted regulatory filings in Japan, Canada and Australia and will initiate additional filings in other countries in 2017.

In addition to the approval, Biogen was also given a rare pediatric disease priority voucher. The company can use it to accelerate the review of another program, or sell it to another company.