Regulatory Updates

The FDA has approved the gene therapies Lyfgenia and Casgevy to treat patients with sickle cell disease. Casgevy is the first FDA-approved gene therapy to use the CRISPR gene editing technology.

If approved, elafibranor would be a second-line treatment for patients with primary biliary cholangitis. The Prescription Drug User Fee Act action date is June 10, 2024.

Augtyro, approved last month, launched with a month wholesale acquisition $29,000 for patients with ROS1-positive non-small cell lung cancer.

Fabhalta was approved to treat patients with paroxysmal nocturnal hemoglobinuria and will have a wholesale acquisition cost of $550,000 per year.

Jaypirca is also approved to treat mantle cell lymphoma. It has a list price of $21,000 for a 30-day supply.

Twice-daily danuglipron was being studied in a phase 2 study in adults with obesity. Pfizer is now conducting a pharmacokinetic study for a once-daily formulation.

The FDA has set a date of July 07, 2024, for Zorvye 0.15% for adults and children six and up with the chronic skin disease atopic dermatitis.

Regulators indicated that an additional trial would be needed to assess the efficacy of reproxalap to treat patients with dry eye. Aldeyra has submitted to the FDA a Special Protocol Assessment for the new trial.

The product, OX124, uses a powder-based technology that provides rapid absorption of naloxone for patients experiencing an opioid overdose. The FDA review date is July 15, 2024.

Ogsiveo will be available within five to 10 business days to treat patients with desmoid tumors. It will launch with a price of $29,000 for a 30-day supply.

Newly launched include generics of Spiriva HandiHaler, Forteo, Livalo and Farxiga.

Janssen is seeking approval for use after Tagrisso in patients with metastatic non-small cell lung cancer.

A regulatory decision will not be made by the Prescription Drug User Fee Act (PDUFA) target action date of Dec. 16, 2023.

Truqap is an AKT kinase inhibitor that, along with Faslodex, reduced the risk of disease progression or death by 50%.

Augtyro will be available in mid-December to treat patients with metastatic ROS1-positive non-small cell lung cancer. It has a list price of $29,000 a month.

Bimzelx is the first dual IL-17 A/F inhibitor to treat moderate-to-severe plaque psoriasis. It launches with a list price of $7,200 per syringe.

Aliqopa was granted accelerated approval in September 2017 to treat adult patients with relapsed follicular lymphoma.

Ixchiq was approved through the FDA Accelerated Approval pathway.

Fruzaqla is the first inhibitor of all three VEGF receptor kinases. The list price is $25,200 for a 28-day supply of a 5 mg dose and $6,300 for a 28-day supply of 1 mg dose

Adzynma is a recombinant protein designed to replace the deficient ADAMTS13 enzyme.

Tirzepatide, with the brand name Zepbound, is expected to be available by the end of the year in six doses at a list price of $1,059.87, which is about 20% lower than semaglutide.

Voquezna is a potassium-competitive acid blocker, which has a mechanism similar to proton pump inhibitors but with a more rapid action. It will be available in December and has a list price of $650 for a 30-count bottle.

Amgen’s Wezlana is approved to treat multiple inflammatory diseases. It is expected to launch no later than Jan. 1, 2025

If approved, mavorixafor would be the first therapy for WHIM syndrome, an ultra-rare disease that can cause recurrent lung infections, papillomavirus-related warts, and an increased risk of developing certain types of cancer. The PDUFA date is April 30, 2024.

Loqtorzi is indicated in combination with chemotherapy for first-line treatment and as monotherapy for patients with disease progression on or after platinum containing chemotherapy.

Agamree is a novel corticosteroid that will be available in the first quarter of 2024.

Mirikizumab — now with the brand name — Omvoh has a list price of $9,593 per month for the IV formulation and $10,360 per subcutaneous administration.

N-803 — with the brand name Anktiva — plus the Bacillus Calmette-Guérin vaccine is being reviewed by the FDA to treat non-muscle-invasive bladder cancer. The goal date is April 23, 2023.

Akebia has resubmitted its NDA for vadadustat with postmarketing safety data from Japan, where it has been on the market for more than three years. The new review date is March 27, 2024.