Each month, we round up the latest FDA approvals and regulations to give you an expert take on what you need to know.
FDA Approval for First-of-Kind Therapy
In early August, the FDA approved Onpattro (patisiran) for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients.1
hATTR is a rare genetic disease which causes buildup of proteins in nerves and organs. This condition is found in less than 5,000 people in the United States. Estimates suggest this therapy will cost between $350,000 and $450,000 annually. This is an infused product so this cost will likely be on the medical benefit vs. the pharmacy benefit. 2
There is much more to this approval, however. This is a new therapeutic category known as small interfering ribonucleic acid (siRNA). This product silences the portion of the RNA responsible for production of the disease-causing proteins.1 This will potentially allow the slowing, stopping, or even reversing of the condition. This technology opens the potential for treatment of other genetic conditions which currently do not have good therapeutic options.
EpiPen in the News
On August 16th, the FDA approved the first generic version of EpiPen and EpiPen Jr (epinephrine) auto-injector for the emergency treatment of allergic reactions, including those that are life-threatening (anaphylaxis), in adults and pediatric patients.3 This is great news as back to school time generally sees a spike in prescriptions for EpiPens (having an extra pen for school, replacing expired pens, etc...). Unfortunately, we still do not know when the Teva Pharmaceuticals generic version will be on pharmacy shelves or what the cost will be once available.4
The FDA then announced on August 21, it is extending the expiration dates of many batches of EpiPen. This is designed to help address shortages not requiring patients to get a refill due to expired product.5 Hopefully, this new stability data will be applied moving forward to assure the efficacy of older EpiPens and delay the need to discard expired EpiPens.