Catalyst Pharmaceuticals (NASDAQ:CPRX) is expecting the FDA to rule on its marketing application for Firdapse, a form of 3,4-diaminopyridine used for the treatment of Lambert Myasthenic Syndrome (LEMS), on or about November 28. The so-called Prescription Drug User Fee Act (PDUFA) date isn’t quite a deadline, but a vast majority of marketing application decisions are made on or before their PDUFA dates. If approved, the drug should lead to significant improvements in safety or effectiveness of the treatment, or improvements in prevention or diagnosis of the condition.
Today, ahead of the big event, Catalyst hosted a quarterly call in which questions centered on potential near-term Firdapse approval, as well as go-to-market strategy, including patient access and pricing. Cantor analyst Charles Duncan came away from the call with greater confidence in the approvability of Firdapse.
Duncan noted, “We are confident of Firdapse’s approvability and likely appropriate orphan disease pricing commensurate with the value that an approved, clinically validated drug should do. Today we believe that Catalyst has made progress on a deep-diligence commercial strategy that makes it well-positioned for a solid launch.”
The analyst continued, “Looking back over 2018, we see the most important progress from Catalyst as the successful NDA filing for Firdapse in LEMS and its efforts in building out a commercial base including senior hires and a sales team of ~20. In the next few months, we see the most important upcoming catalysts as Firdapse approval and announcement of pricing. Management is confident that the value brought by an approval, particularly in expanding patient access, will justify orphan pricing appropriate for the market size of LEMS. We think Firdapse can command pricing similar to what is seen with other recent neuromuscular approvals in the $160-500K range. We believe that Catalyst Pathways, the company’s patient assistance/access program, is well-designed based on precedent programs and will allow smooth transition of the ~300 patients currently on the free drug and that all patients will benefit from affordable access with minimal copays.”
Net-net, Duncan reiterates a Buy rating on CPRX shares, with a price target of $8.00, which represents a potential upside of 163% from where the stock is currently trading. (To watch Duncan’s track record, click here)
Wall Street’s confidence on the drug maker speaks for itself; CPRX has received a whopping 5 buy ratings in the last three months. Meanwhile, the $6.60 price target suggests a potential upside of nearly 114% from the current share price. (See CPRX’s price targets and analyst ratings on TipRanks)