FDA approves drug for form of Lou Gehrig’s disease
- Biogen’s drug targets a genetic mutation that affects about 500 people
- It’s an injectable medication that requires maintenance doses every month
- The FDA is requiring additional research to confirm the drug's benefits
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(NewsNation) — U.S. health regulators have approved a first-of-its-kind drug for a rare form of Lou Gehrig’s disease.
The Food and Drug Administration approved tofersen, Biogen’s infused drug for patients with a rare genetic mutation that affects fewer than 500 people in the U.S. The drug is expected to sell under the brand name Qalsody.
“The FDA has granted conditional approval of this medication for a very specific type of a person with ALS that carries a specific type of gene mutation. That accounts for about 2% of ALS cases,” said Dr. Yalda Safai, a psychiatrist and medical doctor.
The drug is the first intended to treat an inherited form of ALS, or amyotrophic lateral sclerosis, which destroys nerve cells needed for basic functions. Tuesday’s approval came despite a company study that failed to meet its goal of significantly slowing the disease.
The drug did make a difference on laboratory measures of ALS. The FDA is requiring additional research to confirm the drug’s benefit.
“I say conditional approval by the FDA because with certain devastating illnesses, when a drug shows promise, the FDA puts it on a fast-track pace to approval,” Safai said. “It’s a conditional approval and requires further testing while this drug is being marketed.”
In September, the FDA granted full approval to another ALS drug based on one small, mid-stage study in which patients appeared to progress more slowly and survive several months longer. Normally, the FDA requires two large studies or one study suggesting a “very persuasive” improvement in survival.
Some insurers have limited access to the new drug, Relyvrio, citing its uncertain benefits and $158,000-per-year cost.
Biogen did not announce a price for its drug Tuesday but said it will be “comparable to other recently launched ALS treatments.”
“It is an injectable formulation with medication that’s injected into the spine and requires maintenance doses every month. The cost is estimated to be pretty high,” Safai said. “Its side effects are minimal. They include fatigue and a little bit of pain.”
The ALS Association and other patient groups hailed the approval.
The Associated Press contributed to this report.
