FDA Updates for the Week of Sept. 12, 2022


The FDA has approved new therapies for psoriasis and kidney dysfunction. The agency has also scheduled an advisory committee meeting for nonprescription birth control and accepted applications for longer-acting aripiprazole, Rett syndrome drug, and an enzyme replacement therapy. Additionally, Clovis has submits supplemental applications for Rubraca.

FDA approves first-in-class therapy for psoriasis.

The FDA has approved Sotyktu (deucravacitinib) to treat adults with moderate-to-severe plaque psoriasis. Developed by Bristol Myers Squibb, Sotyktu is an oral, allosteric tyrosine kinase 2 (TYK2) inhibitor. This is the first approval worldwide of a TYK2 inhibitor.

The approval is based on results from the pivotal phase 3 POETYK PSO-1 and POETYK PSO-2 clinical trials. The trials demonstrated superior efficacy of once-daily Sotyktu compared with placebo and twice-daily Amgen’s Otezla (apremilast) in 1,684 patients aged 18 years and older with moderate-to-severe plaque psoriasis. Responses with Sotyktu persisted through 52 weeks.

FDA approves Terlivaz for kidney dysfunction syndrome.

The FDA has approved Mallinckrodt’s Terlivaz (terlipressin). Terlivaz is the first therapy approved to improve kidney function in patients with hepatorenal syndrome (HRS), a rare form of kidney disfunction. An injection, Terlivaz is approved for adults who have a rapid reduction in kidney function, which is an acute and life-threatening condition requiring hospitalization.

Hepatorenal syndrome is characterized by a progressive deterioration in kidney function in people with advanced liver disease. This is most common in those with advanced cirrhosis and ascites, an abnormal buildup of fluid in the abdomen that is often related to liver disease. The prognosis is very poor. Hepatorenal syndrome involving rapid reduction in kidney function is estimated to affect between 30,000 and 40,000 Americans annually. The only cure is a liver transplantation.

FDA schedules advisory committee meeting for nonprescription birth control.

The FDA has scheduled a joint meeting of the Nonprescription Drugs Advisory Committee (NDAC) and the Obstetrics, Reproductive, and Urologic Drugs Advisory Committee (ORUDAC) on Nov. 18, 2022, to review the Perrigo’s application for Opill daily oral contraceptive for over-the-counter use.

Opill is a progestin-only daily birth control pill, which is also referred to as a mini pill or non-estrogen pill. While progestin-only birth control pills can still increase your risk of blood clots and stroke, they’re generally seen as safer for some women, including those who smoke, have high blood pressure or those at risk for blood clots.

HRA Pharma, a Perrigo company, had submitted its application in July 2022.

FDA accepts NDA for longer-acting aripiprazole.

The FDA has accepted Otsuka’s/Lundbeck’s new drug application (NDA) for aripiprazole to treat schizophrenia in adults and for maintenance monotherapy treatment of bipolar I disorder in adults. The FDA target date for completion of the review is April 27, 2023.

The therapy is a long-acting medication intended for dosing every two months via intramuscular injection. If approved, it will be available in 960 mg and 720 mg prefilled syringes. It will deliver sustained plasma concentrations similar to that demonstrated in studies with aripiprazole once-monthly long-acting injectable. It is intended for the populations as the once-a-month Abilify Maintena (aripiprazole).

The NDA is supported by a 32-week bridging trial in which aripiprazole two-month showed comparable effectiveness and consistent safety profile to aripiprazole one-month. The 960 mg dosage aripiprazole two-month met the primary endpoint criteria establishing similarity of aripiprazole plasma concentrations and thus comparable effectiveness to aripiprazole once-monthly 400 mg over a two-month dosing interval. The therapy was generally safe and well tolerated and patients did not show any new safety concerns compared with aripiprazole once-monthly 400 mg.

FDA accepts NDA for Rett syndrome drug.

The FDA has accepted Acadia Pharmaceuticals’ new drug application (NDA) of trofinetide to treat patients with Rett syndrome. The FDA has granted a priority review and assigned a Prescription Drug User Fee Act action date of March 12, 2023.

Rett syndrome is a neurodevelopmental disorder that affects mostly girls and is caused by mutations on the X chromosome on a gene called MECP2. Children experience normal early growth followed by slowing of development. Gradually, mental and physical symptoms appear, causing progressive loss of motor skills and the ability to communicate. The incidence in the United States is one in 10,000 girls.

FDA accepts BLA for ultra rare enzyme disorder.

The FDA has accepted the biologics license application (BLA) and granted priority review designation for velmanase alfa, enzyme replacement therapy for alpha-mannosidosis, a rare genetic condition characterized by an inability to properly break down complex sugars in the body’s cells. It is caused by mutations of the MAN2B1 gene.

Developed by Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici, velmanase alfa is a recombinant form of human alpha-mannosidase intended to supplement the natural enzyme. The buildup of sugars can affect many organs and systems including the central nervous system. Effects of the disease vary and progress over time, and symptoms can include recurrent chest and ear infections, hearing loss, distinctive facial features, muscle weakness, skeletal and joint abnormalities, visual abnormalities, or cognitive abnormalities. The prevalence of alpha-mannosidosis is estimated to be 1 in every 500,000 people.

The FDA set an action date in the first half of 2023 under the Prescription Drug User Fee Act.

Clovis submits supplemental applications for Rubraca for advanced ovarian cancer.

Clovis Oncology has submitted a supplemental new drug application (sNDA) with the FDA and a Type II variation with the European Medicines Agency (EMA) for approval of Rubraca (rucaparib) as first-line maintenance treatment for women with advanced ovarian cancer regardless of biomarker status.

The submissions are based on positive data from the monotherapy analysis of the phase 3 ATHENA trial. These data demonstrated that Rubraca as first-line maintenance treatment significantly improved investigator-assessed progression-free survival (PFS) compared with placebo in women with advanced ovarian cancer regardless of biomarker status.

The FDA has recommended that the company wait for more mature overall survival data from ATHENA-MONO to submit the sNDA. Without overall survival data, the sNDA may need to be discussed at an Oncologic Drugs Advisory Committee (ODAC) meeting.

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