FDA Drug Approvals

FDA Approved Drugs: June, 2024

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RYTELO

RYTELO is the first and only telomerase inhibitor approved by the U.S. Food and Drug Administration. It is indicated for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring 4 or more red blood cell units over 8 weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESA).

It is a first-in-class treatment that works by inhibiting telomerase enzymatic activity. Telomeres are protective caps at the end of chromosomes that naturally shorten each time a cell divides. In LR-MDS, abnormal bone marrow cells often express the enzyme telomerase, which rebuilds those telomeres, allowing for uncontrolled cell division. Developed and exclusively owned by Geron.

IQIRVO

Iqirvo (elafibranor) is a first-in-class oral, once-daily peroxisome proliferator-activated receptor (PPAR) agonist. Iqirvo was in-licensed from GENFIT in 2021. The accelerated approval of Iqirvo is based on data from the Phase III ELATIVE trial published in the New England Journal of Medicine.

SOFDRA

Sofdra (sofpironium) gel, 12.45%, is now the first and only chemical entity approved for excessive underarm sweating (primary axillary hyperhidrosis). The treatment is indicated for usage in adults and children aged nine years and above. The FDA’s decision was based on results from the two Phase III CARDIGAN studies in 701 patients.

PIASKY

PiaSky (crovalimab-akkz) is the first monthly subcutaneous (SC) treatment for paroxysmal nocturnal haemoglobinuria (PNH). Additionally, with the option to self-administer, PiaSky may provide an alternative to existing intravenous (IV) C5 inhibitors, potentially helping to reduce treatment burden. The recommendation is based on the COMMODORE 2 study results, where SC PiaSky given every month demonstrated equivalent disease control and comparable safety to IV eculizumab given every two weeks.

OHTUVAYRE

Ohtuvayre (ensifentrine) is the first inhaled therapy for the maintenance treatment of COPD that combines bronchodilator and non-steroidal anti-inflammatory activities in one molecule. Verona has evaluated nebulized Ohtuvayre in its Phase 3 clinical program ENHANCE (“Ensifentrine as a Novel inHAled Nebulized COPD thErapy”) for COPD maintenance treatment. Ohtuvayre met the primary endpoint in both ENHANCE-1 and ENHANCE-2, demonstrating statistically significant and clinically meaningful improvements in lung function. A fixed-dose combination of ensifentrine and glycopyrrolate, a LAMA, is currently under development for the maintenance treatment of COPD. Ensifentrine has potential applications for development in non-cystic fibrosis bronchiectasis, cystic fibrosis, asthma and other respiratory diseases.

FDA Approved Drugs: August, 2023

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Izervay (avacincaptad pegol sodium)

FDA approved Izervay, a complement C5 inhibitor, for the treatment of geographic atrophy secondary to age-related macular degeneration, according to a press release from Astellas Pharma.

The approval was based on the data from the GATHER1 and GATHER2 phase 3 clinical trials in which monthly 2 mg injections of Izervay (avacincaptad pegol intravitreal solution) showed a statistically significant reduction (P < .01) in the rate of geographic atrophy growth at 12 months. The slowing in disease progression was seen as early as 6 months, while up to a 35% reduction in progression occurred in the first year of treatment.

Talvey (talquetamab-tgvs)

FDA granted accelerated approval of Talvey (talquetamab-tgvs), a first-in-class bispecific antibody for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody.

Talvey is a bispecific T-cell engaging antibody that binds to the CD3 receptor on the surface of T cells and G protein-coupled receptor class C group 5 member D (GPRC5D) expressed on the surface of multiple myeloma cells, non-malignant plasma cells and healthy tissue such as epithelial cells in keratinized tissues of the skin and tongue.

Elrexfio (elranatamab-bcmm)

FDA granted accelerated approval to elranatamab-bcmm (Elrexfio, Pfizer, Inc.), a bispecific B-cell maturation antigen (BCMA)-directed CD3 T-cell engager, for adults with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.

Sohonos (palovarotene)

FDA approved Sohonos (palovarotene) capsules as a retinoid indicated for the reduction in volume of new heterotopic ossification in adults and pediatric patients aged 8 years and older for females and 10 years and older for males with fibrodysplasia ossificans progressiva (FOP).

The FDA approval was based on the pivotal efficacy and safety data from the Phase 3 MOVE trial, the first and largest multicenter, open-label trial in adult and pediatric patients.

Veopoz (pozelimab-bbfg)

FDA approved Veopoz (pozelimab-bbfg) for the treatment of adult and pediatric patients 1 year of age and older with CHAPLE disease, also known as CD55-deficient protein-losing enteropathy. Veopoz is the first and only treatment indicated specifically for CHAPLE. Veopoz, a fully human monoclonal antibody, is designed to target complement factor C5, a protein involved in complement system activation.

The FDA approval was based on results from a Phase 2/3 open-label trial that investigated the efficacy and safety of pozelimab in 10 patients aged 3 to 19 (median of 8.5 years).

Eylea HD (aflibercept)

FDA approved EYLEA HD (aflibercept) Injection 8 mg for the treatment of patients with wet age-related macular degeneration (wAMD), diabetic macular edema (DME) and diabetic retinopathy (DR). This is the first and only treatment approved in wAMD and DME for immediate dosing at 8-week and up to 16-week intervals following three initial monthly doses

Approval was based on the pivotal PULSAR and PHOTON trials in which EYLEA HD demonstrated clinically equivalent vision gains to EYLEA (aflibercept) Injection 2 mg that were maintained with fewer injections.

Tyruko (natalizumab-sztn)

FDA approved biosimilar Tyruko (natalizumab-sztn), developed by Polpharma Biologics. Tyruko is approved to treat all indications covered by the reference medicine and is the first and only FDA-approved biosimilar for relapsing forms of multiple sclerosis (MS).

Sandoz entered into a global commercialization agreement for Tyruko with Polpharma Biologics in 2019. Under this agreement, Polpharma Biologics will maintain responsibility for development, manufacturing and supply of the active substance in Tyruko. Through an exclusive global license, Sandoz has the rights to commercialize and distribute it in all markets.

FDA Approved Drugs: November, 2021

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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

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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.