August 8 RxAdvocate July, 2023 — Newsletter
July Stories:
- First Gene Therapy for Hemophilia A
- UV Safety Month
- First Gene Therapy for Muscular Dystrophy
- Employee Highlights
First Gene Therapy for Adults with Severe Hemophilia A
The U.S. Food and Drug Administration (FDA) approved the first one-time gene therapy for adults with severe hemophilia A, Roctavian. This approval comes after an initial rejection in 2020 due to a request for more long-term data to ensure the drug effectiveness did not significantly wane over time.
BioMarin, the manufacturer of Roctavian, is pricing Roctavian at a wholesale acquisition cost of $2.9 million. The Institute of Clinical and Economic Review, a panel of drug pricing experts, found the drug would be cost effective at around $2 million per treatment. The good news is BioMarin is setting up an outcomes-based warranty program designed to reimburse payers up to the full cost if Roctavian does not meet treatment expectations.
Hemophilia A affects about 1 in 10,000 people; however, Roctavian is only approved to treat those with severe disease. BioMarin estimates 6,500 adults suffer from severe hemophilia A in the U.S., but Roctavian may only be used in those who test negative for the viral vector antibody used to deliver the gene therapy. Due to this negative antibody test requirement, the company expects only about 2,500 patients will be eligible to receive Roctavian.
Additionally, unlike other gene therapies, Roctavian is treating a disease with various therapeutic options; therefore, analysts are predicting the initial sales ramp will be slow. Click Here to Read More.
UV Safety Awareness Month
Although July is UV Awareness Month, sun safety is always in season! Skin Cancer is the most common Cancer in the US. According to the FDA in 2023, 97,610 new melanomas will be diagnosed (about 58,120 in men and 39,490 in women). And at least 1 in 5 Americans will develop Skin Cancer by the age of 70. Too much sun exposure can result in sunburns, early skin aging and skin cancer.
Helpful tips for prevention:
- Limit your time in the sun, especially between 10:00 AM – 2:00 PM.
- FDA recommends wearing a SPF value of 15 or higher. Check FDA’s website for recent sunscreen brand recalls (Banana Boat, Neutrogena Aveeno, Family Dollar Products)
- FDA recommends “Broad Spectrum” sunscreen products only. Broad spectrum provides protection against the two types of UV radiation, UVA and UVB.
- Wear clothing or hats to cover your sun exposed skin. Including when riding in cars, airplanes, bus, etc.
- Choose sunglasses with a high UV rating to protect your eyes.
- Say no to tanning beds. UV radiation amounts are 10 to 15 times higher than the sun at its peak intensity.
- Perform monthly skin self-exams and see a dermatologist annually.
First Gene Therapy for Duchenne Muscular Dystrophy is Here
First Gene Therapy for Duchenne Muscular Dystrophy is Here
Duchenne muscular dystrophy (DMD) had a grim treatment approach prior to June 22, 2023. Traditionally, DMD treatment only addressed the symptoms of DMD; however, there was no therapy that focused on the defective gene itself. Decades of perseverance, devotion, and research led to the development and recent FDA approval of Elevidys—the first and only gene therapy for treatment of certain patients with DMD. Elevidys is designed to deliver an encoding gene into the body’s muscles leading to the production of dystrophin protein which is present in normal muscle cells. Elevidys is administered only once as an intravenous infusion.
Duchenne muscular dystrophy (DMD) is a rare, and fatal genetic condition that’s caused by a defective gene resulting in the absence of dystrophin, a protein that’s necessary to keep muscle cells intact. The lack of dystrophin leads to difficulty walking, learning disabilities, heart issues, and breathing problems due to the weakening of muscles involved in heart and lung function. Symptoms associated with DMD typically begin between 3 to 6 years of age, and mainly affect males. Approximately 1 in every 3,300 boys are affected by this disease. Due to heart and/or respiratory failure, patients with DMD often don’t survive past their twenties.
Elevidys has a flat WAC price of $3.2M, which will be consistent for all patients regardless of weight or number of vials required for treatment. Since Elevidys will likely be part of the medical benefit, it is imperative that groups work with their consulting teams and vendors regarding coverage updates. Source Source
Employee Highlights!
Employee Highlights!
We are excited to announce the promotion of Richard Lo to the Director of Clinical Services!
Thank you, Richard, for all of your hard work and dedication!
Please welcome new hire, Domingo Esparza, our newest Data Analyst!
Please welcome new hire, Conrad Hall, our newest Data Analyst!
Contact us
Visit us on the web at www.rxconnectionllc.com.
If you would like additional information on our services please contact:
Orlando Neal — Principal
Orlando.Neal@rxconnectionllc.com
Orlando.Neal@rxconnectionllc.com