On Dec. 12, 2019, the U.S. Food and Drug Administration (FDA) granted accelerated approval to Vyondys 53™ (golodirsen) intravenous injection as the first treatment for a specific type of Duchenne muscular dystrophy (DMD). DMD is a rare genetic disorder associated with muscle degeneration and is caused by the absence of a protein known as dystrophin. While the initial clinical study of Vyondys 53™ demonstrated improvement in protein (dystrophin) levels, the FDA is requiring additional trials be conducted to establish further clinical benefits. In Europe and North America, the prevalence of DMD is approximately 6 per 100,000 individuals, according to the Muscular Dystrophy Association.
On Dec. 11, 2019, the U.S. FDA announced it had sent warning letters to Alkermes, Inc. for the misbranding of an extended-release injection of naltrexone, branded as Vivitrol®. Vivitrol® is approved for the prevention of relapsing opioid dependence following opioid detoxification and is considered an effective treatment for opioid use disorder (OUD). The warning letters were issued in relation to printed advertisements for Vivitrol® that failed to sufficiently communicate important warnings and precautions listed in its approved labeling, including the risks associated with reduction in opioid tolerance associated with Vivitrol® treatment. Decreased tolerance may lead to the increased potential for an overdose if an opioid medication is taken at pre-treatment doses. The FDA has requested that Alkermes, Inc. stop all advertising practices that misbrand Vivitrol® and provide a plan of action to distribute truthful and corrective messages to individuals that received misleading promotional materials.
On Dec. 11, 2019, The BMJ released results of a clinical trial comparing naproxen to low-dose colchicine as treatments for gout flares in a primary care setting. Gout, a form of inflammatory arthritis, occurs in about 4% of American adults – or about 6 million men and 2 million women. The study showed that there was substantial improvement in average pain levels over the first seven days for both treatments and no significant difference between the two treatments in average pain reduction. While there were no serious adverse events related to either medication, there were fewer side effects in patients treated with naproxen. The results support consideration of naproxen ahead of colchicine for the treatment of gout flares in patients without contraindications.
New Biosimilar: Avsola™ (infliximab-axxq) Reference Product: Remicade® (infliximab)
Subcutaneous injection for the treatment of adult patients with acute hepatic porphyria; a rare blood disorder [11/20/2019 – Orphan Intravenous solution for the treatment of various auto-immune conditions [12/6/2019 – AMGEN INC]
Novel Drug Approval: Vyondys 53™ (golodirsen)
Subcutaneous injection for the treatment of Duchenne muscular dystrophy (DMD) [12/12/2019 – Accelerated Approval; Fast Track; Orphan Drug; Priority Review – SAREPTA THERAPEUTICS INC]
Novel Formulation: Nouress™ (cysteine hydrochloride)
Intravenous solution approved as an additive to meet nutritional requirements of neonates (one month of age and younger) [12/13/2019 – Priority Review – AVADEL LEGACY]
Indication: Intestinal ulcers
Dosage Form/Strength: 1GM/10ML oral suspension
Average Wholesale Price (AWP): Generic = $682 | Brand = $718
Nebupent® (pentamidine isethionate)
Indication: Prevention of pneumonia in high-risk, HIV-infected patients
Dosage Form/Strength: 300MG = vial for inhalation
Average Wholesale Price (AWP): Generic = $181 | Brand = $200