Global Courant 2023-04-26 01:49:02
Food and Drug Administration regulators approved a first-of-a-kind drug for a rare form of Lou Gehrig’s disease on Tuesday, though they need further research to confirm it actually helps patients.
The FDA has approved Biogen’s injectable drug for patients with a rare genetic mutation estimated to affect fewer than 500 people in the US. It is the first drug for an inherited form of ALS, or amyotrophic lateral sclerosis, a deadly disease that destroys nerve cells needed for basic functions such as walking, talking and swallowing.
Approval came through the FDA’s fast-track, which allows drugs to be launched based on promising early results, before they are confirmed to benefit patients. That shortcut has come under increasing scrutiny from government watchdogs and congressional investigators.
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The FDA is requiring Biogen to continue studying the drug in a trial involving people who carry the genetic mutation but do not yet have ALS symptoms.
ALS patients hope the decision can lay the groundwork for faster approvals to fight the disease, which affects 16,000 to 32,000 people in the US. The FDA has long used expedited approval to accelerate the availability of drugs for cancer and other deadly conditions.
The drug, tofersen, is designed to block the genetic messengers that produce a toxic form of protein that is believed to cause disease in about 2% of ALS patients. Cambridge, Massachusetts-based Biogen will sell it under the brand name Qalsody. Patients receive three initial spinal injections of the drug over a two-week period, followed by a monthly dose. The most common side effects of the drug were pain, fatigue, and increased spinal fluid.
Biogen’s 100-person study failed to show that the drug significantly slowed the disease compared to a sham treatment. Patients were followed for more than six months using a scale that measures declines in basic movements, including writing, walking and climbing stairs.
But those given tophersen showed significant changes in levels of the toxic protein and a second neurological chemical that is considered an important indicator of disease progression.
The FDA on Tuesday approved tofersen, a drug designed to treat a rare, inherited form of ALS, commonly known as Lou Gehrig’s disease. It will be sold under the brand name Qalsody. (Biogen via AP)
“The findings reasonably predict a clinical benefit in patients,” the FDA said in a statement announcing the approval.
Last month, an outside panel of FDA advisers voted unanimously that these changes warranted conditional approval as more data is gathered to confirm the drug’s benefit. The same panel said Biogen’s current data, including the failed patient trial, was not strong enough to warrant full approval.
FDA regulators have the authority to grant expedited approval to drugs that fail to deliver on their anticipated promise, though they rarely exercised that authority until recently. In recent years, the FDA has ramped up efforts to force unproven drugs off the market amid criticism that too many expensive, ineffective drugs remain available for years.
At the same time, the FDA has shown increased “regulatory flexibility” in approving drugs for rare and debilitating neurological diseases, including Alzheimer’s and ALS.
In September, the FDA granted full approval to another ALS drug based on a 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 that suggests a “very convincing” improvement in survival.
Some insurers have limited access to the new drug Relyvrio, citing its uncertain benefit and cost of $158,000 per year.
Biogen did not disclose a price for its drug on Tuesday, but said it “will be comparable to other recently launched ALS treatments.”
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The ALS Association and other patient groups welcomed the approval.
“This is the second time in less than a year that our community has celebrated the approval of a new drug for the treatment of ALS and we are hopeful for the future,” said Calaneet Balas, the group’s president and CEO.
Since Relyvrio’s approval last year, ALS patients and advocates have continued to push the FDA to consider more treatments for the disease. That includes an experimental stem cell treatment from small drugmaker Brainstorm Cell Therapeutics.
In a rare move, the FDA recently agreed to hold a public meeting about the treatment, despite previously refusing to consider the company’s application, citing failed results from its lead study.
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The FDA has now approved four drugs for ALS, only one of which has been shown to extend life. The disease gradually destroys nerve connections necessary for basic movements and – eventually – breathing. There is no cure and most people die within three to five years of diagnosis.
