US FDA approves drug for Lou Gehrig disease

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The United States Food and Drug Administration on Tuesday approved a first-of-a-kind drug for a rare form of Lou Gehrig’s disease.

Although further research is  still carried out, the FDA approved Biogen’s injectable drug for patients with a rare genetic mutation that’s estimated to affect less than 500 people in the U.S. It’s the first drug for an inherited form of ALS.

The Lou Gehrig’s disease also known as the amyotrophic lateral sclerosis (ALS) is a nervous system disease that weakens muscles and impacts physical function. Although the cause is unknown, nerve cells break down and it reduces functionality in the muscles that they supply – basic functions like walking, talking, and swallowing.

 

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Approval came via FDA’s accelerated pathway, which allows drugs to launch based on promising early results, before they’re confirmed to benefit patients. That shortcut has come under increasing scrutiny from government watchdogs and congressional investigators. The FDA is requiring Biogen to continue studying the drug in a trial of people who carry the genetic mutation but do not yet have ALS symptoms.

 

 

ALS patients hope the decision could lay the groundwork for more expedited approvals to fight the disease, which affects 16,000 to 32,000 people in the U.S. The FDA has long used accelerated approval to speed 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 thought to drive the 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 linked to 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 with a dummy treatment. Patients were tracked for more than six months using a scale that measures the decline of basic movements, including writing, walking and climbing stairs.

 

But those who received tofersen showed significant changes in levels of the toxic protein and a second neurological chemical that is considered a key indicator of the disease’s progression.

“The findings are reasonably likely to predict a clinical benefit in patients,” the FDA said in a statement announcing the approval.

 

 

AP/S.S

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