FDA Approves Eight Novel Drugs in August

FDA Approval

The U.S. Food and Drug Administration (FDA) has had a busy August. In addition to the number of draft guidances the agency issued during the month, the FDA has also been busy approving a number of new treatments for a variety of diseases.

The FDA gave the go-ahead to eight novel drugs in August. The FDA also approved a number of other drugs during the month that are under different classifications. Novel drugs are classified as new molecular entities. In its classification, the FDA said: “many of these products contain active moieties that have not been approved by FDA previously, either as a single ingredient drug or as part of a combination product.”

The first novel drug to gain approval was for Japan-based Kyowa Kirin. The FDA approved Poteligeo (mogamulizumab-kpkc) injection for intravenous use for the treatment of adult patients with relapsed or refractory mycosis fungoides or Sézary syndrome, two types of non-Hodgkin lymphoma. The treatment is intended for patients who have had at least one prior systemic therapy.

Alnylam’s Onpattro (patisiran) was approved Aug. 10 for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. The FDA gave the greenlight for this revolutionary treatment on Aug. 10. Not only was Onpattro the first novel drug of August, it is the first and only RNA interference (RNAi) therapeutic to ever be approved. The drug was approved with a priority review status. RNAi is designed to prevent the production of problematic proteins that are the basis for many diseases.

Also on Aug. 10, the FDA gave the green light for New Jersey-based Amicus Therapeutics Galafold (migalastat), the first oral medication for the treatment of adults with Fabry disease. Fabry disease is a rare and serious genetic disease that results from the buildup of a type of fat called globotriaosylceramide (GL-3) in blood vessels, the kidneys, the heart, the nerves and other organs.

The FDA also approved TherapeuticsMD’s Annovera on Aug. 10. Annovera (segesterone acetate/ethinyl estradiol vaginal system) is the first long-acting prescription birth control that is patient-controlled, procedure-free and reversible, according to the company. Annovera is a small, soft flexible ring that prevents ovulation for an entire year (13 cycles) and can be inserted and removed by a woman at her discretion in repeated four-week cycles

On Aug. 20, France-based Biocodex snagged approval for Diacomit (stiripentol) for the treatment of seizures associated with Dravet syndrome in patients two years of age and older who are already taking clobazam. Dravet syndrome is an early onset epileptic syndrome that is thought to affect approximately 2,000 to 8,000 patients in the U.S.

On Aug. 23, Italy-based Dompe secured approval for Oxervate, a treatment for eurotrophic keratitis (NK), a rare and progressive eye disease that can lead to corneal scarring and vision loss. The rare orphan disease affects fewer than 65,000 people in the United States. The disease results from impaired function of corneal nerves. Oxervate, an ophthalmic solution, is based on cenegermin-bkbj, a novel recombinant human nerve growth factor (rhNGF) that is structurally identical to the nerve growth factor (NGF) protein that is made in the human body, including in the ocular tissues, the company said.

Also on Aug. 23, Shire plc snagged approval for Takhzyro (lanadelumab-flyo), a prophylaxis to prevent attacks of hereditary angioedema (HAE) in patients 12 years of age and older. Takhzyro was approved based off multiple late-stage trials that demonstrated efficacy in treating HAE, a rare, genetic disorder estimated to affect about one in 10,000 to one in 50,000 people worldwide. The condition results in recurrent, localized edema (swelling).

The latest novel drug approved by the FDA was late Aug. 30. Merck snagged approval for HIV-1 treatment Pifeltro, a new non-nucleoside reverse transcriptase inhibitor. Pifeltro was approved alongside another Merck HIV treatment, Delstrigo, a once-per-day triple combination treatment. Merck anticipates both drugs being available to wholesalers within a month.

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