With a substantial discount to Catalyst's Firdapse, is Jacobus poised to win physician, payer support for off-label adult LEMS use?
Weeks ago, the FDA endorsed a Lambert-Eaton myasthenic syndrome (LEMS) drug from family-run New Jersey-based company called Jacobus Pharmaceuticals in pediatric patients, on the basis of adult data, to the surprise of Catalyst Pharmaceuticals which only last year got its pricey — yet similar — treatment for the rare autoimmune disorder across the finish line in adults. It has now been revealed that Jacobus’ version carries a price that is less than half of Catalyst’s Firdapse — a move that could fuel off-label prescription in adults.
Before Catalyst’s Firdapse was sanctioned for use by the FDA, hundreds of patients had been able to access a similar drug from compounding pharmacies for a fraction of the cost, or Jacobus’ for free, as part of an FDA-ratified compassionate use program. But the approval of the Catalyst drug, accompanied by market exclusivity spanning seven years — effectively precluded Jacobus and compounding pharmacies from selling their versions.
Dosing for any of these treatments is based on the patient’s weight and disease severity. Catalyst’s Firdapse, which is approved with a maximum dose of 80 mg, carries an average list price of $375,000 a year. The company does not disclose a per pill number, a Catalyst spokesperson told Endpoints News.
The list price for Ruzurgi is $80 for each 10 mg tablet, and Jacobus’ treatment is approved up to a maximum dose of 100 mg, Laura Jacobus, who runs the privately-held company, told Endpoints News. “As an example, the wholesale cost for a 60 mg dosing regimen would be $175,200.00 annually. The cost to support a patient requiring a daily dose of 100 mg would be $292,000.00 annually.”
Vermont Senator Bernie Sanders — ahead of his announcement to make a second attempt at the presidency — spotlighted Catalyst for the “immoral exploitation of patients”. Other critics of Catalyst’s pricing strategy suggested that depending on how Jacobus prices its drug, Ruzurgi, insurers could be persuaded to favor it over Catalyst’s product despite being officially approved for pediatric use only. As far as the FDA is concerned, doctors can prescribe drugs for off-label use when they judge that it is medically appropriate for their patient.
Firdapse landed on the US market this January, and Catalyst has noted it has encountered minimal pushback from payers, and indicated that typically, covered patients pay less than $10 per month out-of-pocket.
In a note published last month, SunTrust Robinson Humphrey’s Edward Nash suggested that despite suggestions to the contrary, “(W)e have not seen any precedent where payers cover an off-label drug for use in unapproved patient population”.
Meanwhile, HC Wainwright’s Andrew Fein suggested that Jacobus, despite the reputation of a “modern-day Robin Hood” is not equipped with the infrastructure nor the experience to support a commercial push of Ruzurgi, even with the approval in hand.
“Handicapped by legal reasons, we do not believe that Jacobus can openly promote off-label use in LEMS adults…It is unclear if and how Jacobus would be able to expand commercial penetration other than through promoting a proliferation of off-label use at academic centers that were enrolled in the compassionate program (which mounts to approximately 200 patients),” he wrote in a May note.
In LEMS patients, the body’s own immune system launches an attack on the neuromuscular junction — which connects nerves and muscles. The condition can associated with other autoimmune diseases, but tends to occurs in patients with cancer. Its prevalence in pediatric patients is not known, but globally it is estimated to affect three per million individuals, according to the FDA. In a recent investor presentation Catalyst suggested there are 3,000 LEMS US patients, of which 300 are on Firdapse.
A STAT report published on Monday suggested that some adult LEMS patients have insinuated that Firdapse is not effective enough and more tablets beyond the approved 80 mg dose must be taken for relief — these extra pills saddle them with out-of-pocket bills that could climb to thousands of dollars monthly. The Catalyst spokesperson did not provide comment on this assertion.