Firdapse, a formerly inexpensive drug that treats a rare autoimmune disorder, received a new annual $375,000 price tag in December, prompting one patient to begin rationing the medication that enables her to walk, according to a CNN report.
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.
Although it was the first therapy for this disease approved in the U.S., patients had been using an unapproved version. The drug uses a chemical called 3,4-DAP, which was found to improve the symptoms of LEMS in 1980. Patients had received the off-label drug for free from Jacobus Pharmaceuticals or found it at a compounding pharmacy for about $300 to $500 per month, or between $3,600 and $6,000 per year.
Bhanu Patel, who has LEMS, said the disease attacks her hip muscles, abdominal muscles and back muscles, making attempts to stand up excruciating. When her physician prescribed 3,4-DAP, she said it changed her life by allowing her to regain basic function.
"Without this medication, you just can't even move" Ms. Patel said.
Ms. Patel had received the medication for free through a specialty program. However, once Catalyst Pharmaceuticals received approval for Firdapse, she was unable to get it free.
In an effort to prevent burdening her family with exorbitant medical bills, she's begun rationing her medication. Instead of taking four pills a day as recommended, she is taking two. She told CNN that she is trying to stretch her three-month supply for as long as possible.
Her son, Krishan Patel, said his mother has been rejected for coverage of the drug, which raises concern that the family may be stuck with a massive bill. She is appealing for coverage as an exception and has also applied with the Assistance Fund, a nonprofit organization that helps pay for patients' copays, but hasn't heard back.
"My mother's livelihood is fundamentally at the hands of a small outfit with full capability to do whatever they want," Krishan said. "You're leveraging human suffering to make money — and that is a heartbreaking idea."
Catalyst has defended the $375,000 price tag, arguing it is in line with similar products in the industry.
Read the full report here.