- Therapy that treats PKAN tvailable under 'compassionate use' regulations.
- Therapy was not granted expedited status from FDA last month.
- Company fulfills near-term promise.
After receiving disappointing news regarding a medicine to treat Pantothenate Kinase-Associated Neurodegeneration (PKAN) a month ago, Retrophin (NASDAQ:RTRX) announced today that the company is moving forward with treatment via a compassionate need regulation. This will make RE-024 available to patients in need in the very near term, fulfilling a promise made by the company a month ago.
RE-024 is an orphan drug that can potentially give critically ill patients much needed relief. As an orphan drug RE-024 had potential to receive expedited clinical trials and studies to fast track FDA approval and by extension, patient treatment.
The news announced today demonstrates that Retrophin is committed to the drug and its eventual approval. RE-024 is a replacement therapy for phosphopantothenate, the reaction product and substrate that is missing in patients with PKAN. Last week a European health regulator approved use of RE-024 in named patients. The company expects 15 patients to be included in the treatment. PKAN is thought to impact between 5,000 and 10,000 patients world wide. The condition typically manifests itself prior to the age of 10. Those that have PKAN typically pass away within 10 years of diagnosis.
The business strategy of Retrophin essentially hinges on orphan drugs. While the patient pool is not typically large with orphan drugs, the treatment options can be quite lucrative because of their rarity. Orphan drugs can also receive an expedited approval process, making them potentially get to the market much faster.
Retrophin markets Chenodal® and Vecamyl®, and its pipeline includes compounds for several catastrophic diseases, including focal segmental glomerulosclerosis (FSGS), pantothenate kinase-associated neurodegeneration (PKAN), schizophrenia, autism, infantile spasms, nephrotic syndrome and others. Retrophin intends to reintroduce Syntocinon Nasal Spray in the U.S. to assist initial postpartum milk ejection.
The equity has been quite volatile in recent weeks after a substantial rise from a year ago. It has a 52 week range of $4.50 to $24.25 and currently trades at about $11.50. The equity began a very strong move in January of this year and peaked a month ago. Today's announcement is the first big news since the decline from the peak.
From an investment standpoint this is an interesting play. The pause in upward trajectory and decline occurred just after the FDA made its decision public. The news today could reinvigorate the equity. Clearly there is an intention by Retrophin management to move forward. At current levels this equity could be very attractive. This is particularly true if the patients in the "name patient" treatment program progress well. Stay Tuned!