FDA Reverses Course and Grants ALS Patients “Compassionate Use” Access to IPLEX

By Kurt R. Karst –      

Earlier this week, FDA announced that the Agency will reverse course and grant Amyotrophic Lateral Sclerosis (“ALS”) patients “compassionate use” access to IPLEX (mecasermin rinfabate [rDNA origin] injection).  FDC Act § 561, which was added to the statute in 1997 by the FDA Modernization Act, identifies the criteria for determining whether an individual patient should have access to an investigational drug for treatment use – that is, a single-patient IND.  FDA approved IPLEX in December 2005 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; however, Insmed Inc., which holds the IPLEX NDA, discontinued marketing the drug as the result of a patent infringement settlement. 

FDA’s decision to grant ALS patients access to IPLEX comes on the heels of a series of letters issued in January 2009 in which the Agency claimed that there was insufficient evidence of the safety and efficacy of IPLEX in treating ALS patients.  The Washington Legal Foundation (“WLF”) announced earlier this month that it had appealed (here and here) those decisions, and “pledged to pursue relief in the courts if its appeals are denied.” 

According to FDA’s announcement, “[a]s FDA continued to receive requests for access to Iplex after the initial denials of the single-patient INDs, we continued to evaluate the data and pursued several actions in parallel,” and “the data were useful in that no serious, immediate drug-related toxicities were apparent.”  Under an agreement between FDA and Insmed, access to IPLEX for investigational use in ALS patients will occur in two ways under 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.

According to a WLF press release, “[a]lthough FDA did not explain how it would conduct its lottery for ALS patients who have not yet filed Treatment INDs, the system appears to be designed to ensure that FDA can obtain meaningful data regarding Iplex’s safety and effectiveness.”