By Dalia Deak
The latest development in the race for approval between Jacobus Pharmaceutical Company and Catalyst Pharmaceuticals is a ‘refuse to file’ letter that the FDA issued to Catalyst indicating that Catalyst’s New Drug Application for Firdapse was incomplete. Both companies are competing for approval of slightly modified forms of a drug—3,4-diaminopyridine, or 3,4-DAP— to treat Lambert-Eaton myasthenic syndrome (LEMS). The winner will receive 7 years of exclusive marketing rights to the drug.
LEMS is an autoimmune disorder that affects an estimated 3,000 people in the United States. It is a rare, debilitating disorder that is marked by progressive weakening of the muscles that often begins in young adulthood. The drug in question was initially discovered in the 1970s in Scotland, with researchers in Sweden demonstrating its use in LEMS patients in the 1980s. Jacobus Pharmaceutical Company has been providing a free base form of the drug to patients with a LEMS diagnosis since the early 1990s at no cost (with the exception of postage), though the drug had never received FDA approval.
This distribution was done through the company’s compassionate use program. Compassionate use, or expanded access, is a mechanism by which an investigational drug (i.e., one that has not been approved) is made available for use outside of a clinical trial. This often requires that certain doctors fill out a great deal of paperwork for each patient, a process that can sometimes necessitate patients travel extensively to reach doctors willing to do so or familiar with the process. Alternately, patients diagnosed with LEMS can obtain the drug through a compounding pharmacy, though there are challenges associated with that as well.
That is, this was the case until Catalyst Pharmaceuticals came on the scene. Catalyst applied for approval of a modified version of the drug that does not require refrigeration. Additionally, Catalyst also applied for and received orphan drug status and was granted breakthrough designation. In addition to the 7-year market exclusivity, orphan drug status affords tax credits on clinical research, exemption/waivers on filing fees, and assistance throughout the process. A breakthrough designation indicates that the FDA will expedite the development and review of such drug.
In a presentation to investors, Catalyst indicated that it hopes to generate between $300 and $900 million per year from the drug for treatment of patients with LEMS, and potentially congenital myasthenic syndrome (CMS). The dramatic price increase that this would require (with some estimating that the drug could cost $100,000) has patients and providers worried, so much so that a group of neuromuscular doctors penned an editorial arguing that Catalyst is seeking profits from mostly old medical research. Others have argued that approval of the product, which Catalyst has sought to spend the time and money doing, will allow for doctors everywhere to be able to write prescriptions. It is important to note that the FDA does not take price into account when making decisions regarding the approval of drugs, though some have argued that it should.
It seems that this week’s letter could be a setback for Catalyst, though it is not yet clear how much of an edge it will give Jacobus. Importantly, as the issue of drug pricing continues to be a prominent one during this election cycle, the race to approval for this old drug, and the subsequent price that the winning company sets, may have important implications on a larger scale.