ALS DRUG TO RECEIVE EXPERT SCRUTINY — The FDA’s external panel of neurological experts will discuss tofersen, a novel therapy from Biogen for the treatment of amyotrophic lateral sclerosis, on Wednesday. Tofersen is a spinal-cord injection for patients with a form of ALS caused by a mutation that leads the body to produce toxic proteins. The drug candidate stops the body from making those deformed proteins, which lead to progressive muscle weakness and death. Biogen asked the FDA to grant tofersen accelerated approval pathway last July. The pathway lets the agency consider whether a drug affects a surrogate, or different, endpoint — in this case, a protein called neurofilament light chain — instead of concrete evidence of patient benefit. After receiving accelerated approval, drug manufacturers must conduct an additional trial showing their product works. No one debates ALS’ unmet need: The disease is fatal typically within three to five years from symptom onset. The handful of existing treatments for ALS targets patients’ quality of life but not the causes of the disease. Recent scrutiny: The accelerated approval pathway has been the subject of lawmaker and public scrutiny in recent months. Some argue the pathway puts expensive drugs on the market that don’t help patients and the FDA doesn’t do enough to ensure follow-up trials are completed expeditiously. Disease advocates argue the pathway is crucial for those with rare, fatal diseases — a sentiment Peter Marks, head of the FDA’s Center for Biologics Evaluation and Research, echoed to attendees of the annual Muscular Dystrophy Association meeting on Monday. Marks spoke specifically about the pathway’s use for gene therapies that treat conditions affecting fewer than 100 people. ALS affects 5,000 patients annually; the type of ALS that tofersen would treat affects only about 330 people annually. The FDA is expected to make a decision on tofersen by April 25. It doesn’t have to side with the advice of its expert panel, but it usually does. IT'S TUESDAY. WELCOME TO PRESCRIPTION PULSE. It’s Trea Turner’s world and we’re just living in it. Send tweet. Send news and tips to David Lim (dlim@politico.com or @davidalim) or Katherine Ellen Foley (kfoley@politico.com or @katherineefoley). TODAY ON OUR PULSE CHECK PODCAST, host Ben Leonard talks with reporter Rachel Bluth about California’s plan to partner with Civica, a Utah-based generic pharmaceutical company, to start producing California-branded insulin in a bid to make the drug more affordable. The new insulin, which will cost $30 for a 10 mL vial, is awaiting FDA approval.
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