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Investigational New Drug Application Allows Patients with ALS to Iplex

IND's Allow Patients Iplex Access » The Food and Drug Administration is sharing with the public its decision to allow patients with Amyotrophic Lateral Sclerosis, a fatal neurodegenerative disease also known as Lou Gehrig’s Disease, or ALS, to have access to a drug called Iplex under an Investigational New Drug (IND) application. Iplex (mecasermin rinfabate [rDNA origin] injection), is a combination of two substances: human insulin-like growth factor 1 (IGF-1) and human insulin-like growth factor-binding protein-3 (rhIGFBP-3). Iplex is approved by the FDA only for the treatment of growth failure in children with severe primary IGF-1 deficiency (Primary IGFD) or with growth hormone (GH) gene deletion who have developed neutralizing antibodies to GH. The drug is currently not marketed because of a court order related to patent infringement. Iplex is made by Insmed, a biologics company headquartered in Richmond, Virginia.

FDA’s Decision

The FDA and Insmed have agreed that access to Iplex for investigational use in patients with ALS will occur in two ways under Investigational New Drug applications (INDs):

1. Single-patient INDs requesting “compassionate use” of Iplex for treatment of named patients with ALS, received and date-stamped by FDA’s document room by close of business on March 6, 2009, will be allowed to proceed, and Insmed has agreed to supply Iplex to those patients; and

2. The remaining supply of Iplex, which is very limited, will be used by Insmed to conduct a clinical trial under an IND in which other patients with ALS who are interested in receiving Iplex treatment will be randomly assigned to receive drug through a lottery system.

All patients who receive Iplex under either a single-patient IND or in the Insmed clinical trial must be adequately informed by their treating physician of the possible benefits and risks of the treatment. To facilitate the informed consent process, FDA is making available other documents, as described below, to ensure that healthcare providers and patients have access to more complete information related to the potential risks and benefits of Iplex treatment.

FDA has agreed to allow Insmed to submit a request for cost recovery under existing IND regulations to offset the costs associated with conducting the planned clinical trial. The physicians who submitted the single-patient INDs for “compassionate use” of Iplex will be asked to participate in the data collection for the Insmed clinical trial in order to maximize the collection of data from the very limited supply of drug.

Rationale for FDA’s Decision

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. 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.

FDA has received a number of single-patient IND requests from physicians to allow “compassionate use” of Iplex for named patients with ALS. In reaching its decision on permitting investigational use of Iplex in patients with ALS, FDA recognized that solely granting access to the drug under single-patient INDs would rapidly deplete the limited supply of Iplex and make it virtually impossible to conduct a controlled clinical trial. This is critical, because without adequate controlled clinical trials, it is not be possible to determine whether Iplex is effective, or harmful, in patients with ALS.

The FDA believes its decision represents the fairest way possible to provide access to Iplex, first, because Insmed does not have enough drug for every patient who may request it and, second, because it is important to maximize what can be learned from the remaining supply of drug in case it does have benefit, and could be further developed for widespread use by patients with ALS.

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. These considerations were weighed over the last few weeks by FDA scientists and physicians, who held a series of meetings with Insmed and internal meetings to discuss the best path forward.