Formerly inexpensive drug gets $375K price tag: 8 things to know

Firdapse, an old, formerly inexpensive drug that treats a rare autoimmune disorder, just received a new $375,000 price tag, according to a STAT news report.

Here are eight things to know:

1. In late November, the FDA approved Firdapse as the first treatment for Lambert-Eaton myasthenic syndrome, which affects the connection between nerves and muscles, causing weakness in affected patients.

2. Although it was the first therapy for this disease approved in the U.S., patients have been receiving an unapproved version of this drug, according to the report.

3. In the 1980s, a chemical called 3,4-DAP was found to improve the symptoms of LEMS. Since then it has been used off-label in the U.S. to treat the rare disease. Patients often received the off-label drug for free from Jacobus Pharmaceuticals.When patients receive the chemical from a compounding pharmacy, it costs about $300 to $500 per month.

4. The maker of Firdapse, Catalyst Pharmaceuticals, has been working over the last several years to modify the main chemical and secure regulatory approval.  FDA approval came in November, and the drugmaker will also get market exclusivity for the next seven years.

5. "We recognize that the price for medicines and the insurance coverage barriers are of significant concern to patients, physicians, healthcare providers, payers, and policymakers," Patrick McEnany, CEO of Catalyst Pharmaceuticals, told STAT. In addition, the company made a "comprehensive patient insurance navigation and financial assistance program designed so a patient is paying out of pocket a minimal amount of money," Mr. McEnany said.

6. Although the drug was offered for free, it was a somewhat onerous process to get 3,4-DAP, according to the report. Physicians had to tap the FDA to request access under compassionate use laws.

7. Since the approval, compounding pharmacies are prohibited from making and selling the drug to patients.

8. Catalyst Pharmaceuticals said it has been working to ensure all patients with LEMS have access to the drug. In addition, the drugmaker has been working with insures to ensure they will cover most of the cost.

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