It is no secret that Catalyst Pharmaceuticals Inc. (NASDAQ: CPRX) has had issues with the FDA regarding the approval of its lead product candidate called Firdapse. The company is developing the drug as a treatment for a condition known as Lambert-Eaton Myasthenic Syndrome (LEMS).
But its first New Drug Application (NDA) for Firdapse to the FDA was rejected and instead the agency requested for more data before it can review the drug. Despite the setback, Catalyst has repeatedly stated its commitment to see that Firdapse enters the market.
Additional Phase 3 trial
To meet the FDA’s request for more study data, Catalyst Pharmaceuticals Inc. (NASDAQ: CPRX) is in the process of kicking off a short-term Phase 3 study of Firdapse in 2H2016. Perhaps it is important to mention that before decided to go back to the clinics for another round of late-stage trial of Firdapse, Catalyst sought audience with the FDA for clarity on what the agency required in the additional data.
It understood that the FDA wanted to see additional positive results from a well-controlled trial of Firdapse in LEMS patients. Now that Catalyst is clear on what the FDA needs, it feels confident in undertaking the mini Phase 3 trial on Firdapse.
What happens next?
If the additional data that Catalyst Pharmaceuticals Inc. (NASDAQ: CPRX) will submit to the FDA from the extra Phase 3 study on Firdapse is accepted, the company plans to immediately embark on the process of resubmitting NDA for Firdapse. That is something that is anticipated to come in the back half of 2017.
Besides LEMS, Catalyst is looking to expand the label of Firdapse to include other conditions. That should widen the commercial opportunity for the drug. Nevertheless, Catalyst has not provided an estimate of the amount of revenue it expects to generate annually if Firdapse gets approved for LEMS.
In addition to Firdapse, Catalyst has other candidates that it is also trying to push through clinical trials.
Does Catalyst Pharmaceuticals Inc. (NASDAQ: CPRX) have the financial resources?
What is not in doubt is that undertaking another Phase 3 trial on Firdapse will delay the time to market for the product. Additionally, it will cost Catalyst Pharmaceuticals Inc. (NASDAQ: CPRX) more money than it initially anticipated. But the effort is worth it considering the amount that the company has sank into Firdapse program.
At the end of 2Q2016, Catalyst had cash and equivalents totaling $48 million and there was no doubt. The management offered hope that the funds should be sufficient to complete the work around Firdapse.