A new organization of activist doctors have joined in a drug pricing protest. Neuromuscular specialists are targeting a stirring drug, Firdapse. This drug is expected to spin diagnosis of a singular illness from a no-cost try to a costly affair.
Catalyst Pharmaceuticals asked the FDA to approve the med earlier this year, and it’s counting on Firdapse to increase its sales by $300 million to $900 million, a revenue haul that implies a price of up to $100,000 per patient, The New York Times reports.
Firdapse is to some extent an improved version of an old drug–3, 4-DAP–that one U.S. drug maker Jacobus Pharmaceutical is giving away for free. The drug has been used to treat Lambert-Eaton myasthenic syndrome and congenital myasthenic syndromes for 30 years. But the FDA gave Firdapse orphan drug status, which means it’s entitled to 7 years of market exclusivity if approved
Firdapse’s growth and Catalyst’s skeleton for premium pricing have been stirring debate in biotech circles for months. But now, in an essay published in Muscle Nerve, an organization of 80 physicians contend they’re disturbed about “a potentially damaging cost boost of 3, 4-DAP” if and when Catalyst wins a FDA approval.
Patrick J. McEnany, Catalyst’s Chief Executive Officer said, “Catalyst remains extremely focused on the completion of the NDA submission for Firdapse and on the development of other innovative drugs for the treatment of rare neurological and neuromuscular conditions.” Mr. McEnany continued, “However, we view the Sabril project as a complex, high barrier, generic program, in which we can leverage our extensive experience with our version of vigabatrin, enabling us to rapidly develop a product aligned with our neurological and neuromuscular rare disease focus. Furthermore, this product provides us with an opportunity to add additional shareholder value as we build a product portfolio for commercialization.”
Doctors have indicated that Catalyst is using a loophole in the orphan drug law that allows older, existing drugs to win the designation and its exclusivity benefits.
“We recognize that pricing of some FDA-approved drugs under the ODA may be justified by the substantial investor risk and research and development costs incurred by pharmaceutical companies in preclinical and clinical development,” the doctors note in their article. “However, 3,4-DAP is not a newly discovered molecule, nor is it expensive to manufacture; thus, it differs dramatically from some innovative drugs now in or coming to the market. It has been reported to be effective and safe since 1983.”
Doctors have said that the Congress should close that loophole in the Orphan Drug Act and prevent companies like Catalyst that plan to charge premium prices for long-used meds, after spending minimal time, money and effort on readying them for approval.
“The Orphan Drug Act has been turned on its head in recent years,” Henry A. Waxman, the former Democratic congressman who sponsored the ODA, told the Times. “It has created a special status for orphan diseases that offer large potentials for making generous profits.”
Doctors and scientists who’ve studied Firdapse have claimed it to be “still basically the same drug,” and the raw material for it “was a simple chemical.”
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