FDA Approved Drugs: November, 2021

Voxzogo (BioMarin Pharmaceutical)

BioMarin Pharmaceutical’s Voxzogo (vosoritide) is the first FDA approved therapy for children with achondroplasia, an inherited disorder that causes the most common form of dwarfism. With this approval, the FDA also issued a Rare Paediatric Disease Priority Review Voucher (PRV), which confers priority review to a subsequent drug application that would not otherwise qualify for priority review. Voxzogo, a C-type natriuretic peptide (CNP) analog, represents a new class of therapy, which acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.

Livtencity (Takeda)

Takeda’s Livtencity (maribavir) is the first and only treatment for adults and pediatric patients (12 years of age and older and weighing at least 35 kg) with post-transplant cytomegalovirus (CMV) infection/disease that is refractory to treatment (with or without genotypic resistance) with ganciclovir, valganciclovir, cidofovir or foscarnet. This new molecular entity targets CMV at UL97, resulting in inhibition of viral DNA replication, encapsidation, and nuclear egress. It became available for prescription on December 2, 2021.

Cytalux (On Target Laboratories)

On Target Laboratories’s Cytalux (pafolacianine) is the first targeted fluorescent imaging agent indicated as an adjunct for the intraoperative identification of malignant lesions in patients with ovarian cancer. Cytalux, administered by standard IV in as little as one hour before surgery, binds to folate receptors that are overexpressed in most epithelial ovarian cancers and illuminate intraoperatively under near-infrared light.

FDA Approved Drugs: April, 2021

Qelbree (viloxazine extended-release capsules): Supernus Pharmaceuticals, Inc.

FDA approved Qelbree (viloxazine extended-release capsules) for the treatment of attention-deficit hyperactivity disorder (ADHD) in pediatric patients 6 to 17 years of age. Qelbree represents the first novel non-stimulant treatment for ADHD. Supernus Pharmaceuticalsplans to make Qelbree available in the United States in Q2 2021.

Attention deficit hyperactivity disorder (ADHD) is a mental health disorder that can cause above-normal levels of hyperactive and impulsive behaviors. People with ADHD may also have trouble focusing their attention on a single task or sitting still for long periods of time.

The approval of Qelbree is based on the data from an extensive development program consisting of four Phase III clinical trials that studied more than 1000 pediatric patients from the age of 6 to 17 years. In December 2020, the Company announced positive results from a Phase III trial in adult patients with ADHD and plans to submit a supplemental New Drug Application to the FDA for Qelbree in adults in the second half of 2021.

Nextstellis (Drospirenone and Estetrol Tablets): Mayne Pharma

FDA approved Nextstellis (also trade name: Estelle), (3 mg drospirenone [DRSP] and 14.2 mg estetrol [E4] tablets), for the prevention of pregnancy. Nextstellis is the first and only contraceptive pill containing E4, a naturally produced estrogen during pregnancy, which can now be made from a plant source. Mayne Pharma anticipates the commercial launch of Nextstellisby at the end of June 2021.

Nearly 10 million American women use short-acting combination contraceptives (estrogen and progestin). Of these contraceptives, more than 99% contain ethinylestradiol (EE), a synthetic estrogen that binds widely to all estrogen receptors in the body.

The approval is based on the two-phase III clinical studies conducted in 3,725 women, Nextstelliswas shown both safe and effective and met its primary efficacy endpoint of pregnancy prevention while also meeting a variety of secondary endpoints that demonstrated favorable cycle control, bleeding control, safety, and tolerability.

Nextstellis was developed by a Belgian biotech company Mithra Pharmaceuticals. Mithra has signed 10 licensing deals for Estelle with a number of leading women’s health companies covering United States, Europe, Japan, South Korea, ASEAN, Russia, Brazil, Canada, Middle East, North Africa, Southern Africa and Australia. Mayne Pharma has a 20-year exclusive license and supply agreement in the United States and Australia for Nextstellis.

  • In May 2020, Mithra has entered into an exclusive 20-year license and supply agreement with Mayne Pharma for the commercialization of its combined oral contraceptive E4/DRSP (Estelle) in Australia.
  • In October 2019, Mithrahas signed a License and Supply Agreement (LSA) with Mayne Pharma Group for an exclusive license to commercialize Estelle in the US.

As a result of receiving FDA approval for Nextstellis, Mayne Pharma will pay Mithra US$11m in cash and issue 85.8m ordinary Mayne Pharma shares.