FDA Issues Decision for Compassionate Use of Iplex in ALS

March 23, 2009

March 23, 2009 — The US Food and Drug Administration (FDA) has issued a position statement allowing access for patients with amyotrophic lateral sclerosis (ALS) to mecasermin rinfabate [rDNA origin] injection (Iplex, Insmed) under an investigational new drug (IND) application.

Patients will be able to receive the drug either through single-patient INDs requesting compassionate use or via participation in a clinical trial that will use the remaining supply of the drug, described in an FDA release as "very limited."

"The FDA's decision comes after serious consideration of the needs of patients with ALS and the practical limitations posed by the extremely limited supply of the drug," an FDA statement, issued March 10, said. "The agency has carefully reviewed all available studies and data on the potential benefits and risks to patients with ALS, as well as the need to have as fair a plan as possible for allocating the limited supply of the drug among the patients who want to receive it."

The drug is a combination of 2 substances, human insulinlike growth factor 1 (IGF-1) and human insulinlike growth factor-binding protein-3 (rhIGFBP-3), and is approved by the FDA only for the treatment of growth failure in children with severe primary IGF-1 deficiency or with growth-hormone gene deletion who have developed neutralizing antibodies to growth hormone.

The drug is currently not marketed, the statement notes, "because of a court order related to patent infringement."

Investigational use of Iplex will occur in 2 ways under INDs, the FDA statement notes. Single-patient INDs requesting compassionate use of the drug for treatment of named patients with ALS that were already received and date-stamped by the FDA's document room by close of day on March 6, 2009 will be allowed to proceed, and the company has agreed to supply the drug to those patients.

The remaining supply of the drug will be used by Insmed to conduct a clinical trial under an IND in which ALS patients who are interested in receiving treatment will be randomly assigned to receive the drug through a lottery system.

"FDA has 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 in treating patients with ALS," the statement notes.

For further details regarding the clinical trial and the process for enrolment into the lottery, the statement suggests contacting Insmed at (804) 565-3083 or iplex_als@insmed.com.


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