WASHINGTON (Reuters) - U.S. regulators will allow experimental use of Insmed Inc’s drug Iplex for some patients with Lou Gehrig’s disease, the Food and Drug Administration said on Tuesday.
The company also will begin a clinical trial to determine if Iplex helps patients with the fatal neuromuscular disease or if it may be harmful, the FDA said.
A number of doctors had sought permission to use the drug to treat patients with Lou Gehrig’s disease, also known as amyotrophic lateral sclerosis, or ALS, the FDA said. More than 100 ALS patients have received court-ordered treatment with Iplex in Italy.
Iplex is approved for treating a growth hormone deficiency, but is not currently sold because of a court order related to patent infringement, the FDA said. The company is studying Iplex as a potential treatment for muscular dystrophy.
Similar drugs showed early promise for ALS, but later research failed to find a benefit. Some trials suggested patients may be more likely to die with drug treatment compared with a placebo, the FDA said.
The agency said it “attempted to balance the needs of individual patients who are desperately seeking treatment options for this devastating disease with the need to learn if the drug is in fact beneficial, or harmful” for ALS.
ALS causes muscles to weaken and eventually deteriorate. It usually develops in people between the ages of 40 and 60.
With “very limited” supplies, the FDA and Insmed agreed to provide Iplex to specific patients whose requests were received by March 6. Insmed will use the remaining doses to run a clinical trial to test the drug’s risks and benefits. The trial will be open to other ALS patients through a lottery, the FDA said.
“If the drug works in the clinical trial, we would obviously develop it further and seek approval” for ALS, Insmed Chief Executive Geoffrey Allan said in an interview.
Allan declined to say how many patients might be eligible for the drug under the special access program or through the clinical trial.
Insmed shares were up 13 cents at $1.03 in late afternoon trading on Nasdaq.
Reporting by Lisa Richwine; Editing by Gunna Dickson and Andre Grenon