FDA Grants Biogen’s Ultra-Rare ALS Med Accelerated Approval

In light of a recent expert meeting on the future of Biogen’s amyotrophic lateral sclerosis (ALS) therapy, Qalsody, the FDA approved the medication on the accelerated approval pathway to treat the incredibly rare SOD1-ALS. The SOD1-targeting therapy marks the first-ever approved treatment targeting an ALS subtype’s genetic cause.

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Treating A Sparse But Deadly Disease

SOD1-ALS is caused by a mutation in the superoxide dismutase 1 (SOD1) gene. Only about 330 people in the U.S. are diagnosed with SOD1-ALS, making it an extremely rare disease. Even with the limited market size, Biogen saw the potential in Qalsody’s early development with Ionis Pharmaceuticals and worked hard to bring it to the market. 

Biogen designed Qalsody as an antisense oligonucleotide that targets SOD1 mRNA to decrease SOD1 synthesis. As a result of this process, Qalsody reduces plasma neurofilament light (NfL), a blood-based biomarker of nerve injury and neurodegeneration. 

The FDA approved Qalsody based on its ability to reduce NfL but awarded an accelerated approval, forcing Biogen to complete a confirmatory trial to prove its clinical benefit further. Biogen said the ongoing ATLAS Phase 3 trial will serve as the confirmatory trial.

The approval came after the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee met to discuss Qalsody’s value in treating SOD1-ALS. The committee voted unanimously to consider Qalsody for accelerated or conditional approval based on its ability to reduce NfL. As for consideration for traditional approval, however, the committee voted three yes, five no, and one abstention. 

Still, Biogen is taking the accelerated approval in stride, already looking forward to Qalsody’s future in development and commercialization.

Qalsody Going Forward

With an accelerated approval under its belt, Biogen says that it plans to start rolling Qalsody out within as little as a week. The company mentioned that treatment administration times may vary as institutions learn more about the drug. 

During the clinical trial, 108 patients were randomized 2:1 to receive either 100mg doses of Qalsody or a placebo for 28 weeks. Biogen went on to perform an open-label extension study and conducted an interim analysis at 52 weeks. The therapy is administered intrathecally through a spinal injection. Patients receive three initial doses at 14-day intervals before receiving maintenance doses every 28 days. 

President and CEO of Biogen, Christopher A. Viehbacher, said, “For more than a decade, Biogen has been steadfast in our commitment to pursuing treatments for ALS, and I want to thank the scientists as well as the entire ALS community who have all worked tirelessly to bring this first-of-its-kind treatment to people with SOD1-ALS. Today also marks a pivotal moment in ALS research as we gained, for the first time, consensus that neurofilament can be used as a surrogate marker reasonably likely to predict clinical benefit in SOD1-ALS.”

Qalsody’s accelerated approval is a momentous occasion in the ALS treatment space. Despite being confined to a small niche with few patients, the patients in need could benefit tremendously from Biogen’s efforts to develop Qalsody. The company still has its work cut out as it strives to shift Qalsody’s accelerated approval to traditional approval with evidence from the ongoing ATLAS trial.

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Author

Reed Slater