3 New, and High-Cost, Gene Therapies are Expected This Year


High-priced gene therapies challenge payers to provide access. They are watching the products to be approved the first half of this year.

Gene therapies continue to be at the center of excitement about their curative potential. But because of their high costs, they are also the center of discussions about pricing, access, and value. Late last year, the FDA approved two additional gene therapies, including the highest cost ever for a treatment.

Hemgenix, which was approved by the FDA in November 2022 as a one-time treatment for patients with hemophilia B, costs $3.5 million. At the time of its approval, a spokesperson for the therapies manufacturer, CLS Behring, told Formulary Watch that Hemgenix was priced to provide value and will generate cost savings in the form of reduced annual bleed rates and reduced or eliminated use of prophylactic therapy.

Related: Updated: FDA Approves $3.5 million Gene Therapy Hemgenix for Hemophilia B

So far, the FDA has approved seven gene therapies. The most recent is Adstiladrin (nadofaragene firadenovec-vncg), which was approved in December 2022 to treat adult patients with high-risk non-muscle-invasive bladder cancer. It is administered by catheter into the bladder once every three months. The therapy’s manufacturer, Ferring Pharmaceuticals, has not discussed pricing, however.

Langley Kyle, Pharm.D.

Langley Kyle, Pharm.D.

In the first half of this year, three additional gene therapies are expected to be approved, and they likely will be high priced, putting increased pressure on the healthcare system. Gene therapies are covered under the medical benefits of health plans, Langley Kyle, Pharm.D., manager of clinical services at RxBenefits, said at recent webinar sponsored by the company.

“Often times, these are for rare diseases we have no cure for,” she said. “The annual costs for hemophilia can exceed $250,000, so it’s easy to see how a one-time treatment like Hemgenix can combat costs. That can have an impact on treatment and the quality of life as well. But understanding the true impact is going to take time.”

But she said that because the patient populations are small, it’s hard to find objective data to indicate what the long-term benefit of these therapies are.

Employers and health plans are considering several mechanisms to address gene therapies, including risk pools, carve outs, reinsurance, and value-based contracting. Other are coming up with novel ways to address these costs. For example, in January a group of Blue Cross Blue Shield affiliated health plans formed medication contracting company, Synergie Medication Collective, to focus on affordability and access to gene therapies, infusible cancer drugs and other medications administered in a clinical setting. Synergie executives said they plan to use their collective reach to engage with pharmaceutical manufacturers and other industry stakeholders to bring transparency and provide more affordable access to high-cost treatments, including gene therapies and infusible cancer drugs.

Pharmaceutical companies are offering a variety of pay-over-time and value-/outcomes-based programs. One example is Novartis’ Zolgensma (onasemnogene abeparvovec-xioi) to treat spinal muscular atrophy, a progressive disease of the nerve cells. When it was approved in May 2019, it was launched with an option to pay over five years and an option for an outcomes-based agreement.

Spark Therapeutics created a similar program outcomes-based and pay-over time programs for Luxturna (voretigene neparvovec-rzyl), which was approved in 2017 as a one-time gene therapy indicated for the treatment of patients with a rare form of inherited blindness. Spark also has contracted directly with insurers for payments for Luxturna to avoid hospitals having to purchase a very expensive therapy and then wait to be reimbursed by insurers.

2023 Expected Approvals

One of the first of the new approvals could be a one-time infusion for hemophilia A. If approved, BioMarin Pharmaceutical’s Roctavian (valoctocogene roxaparvovec) would be the first gene therapy to launch in the United States for severe hemophilia A. It works by delivering a functional gene that is designed to enable the body to produce Factor VIII on its own without the need for continued hemophilia prophylaxis. It has a PDUFA date is March 31, 2023. Hemophilia A is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Results of a phase 3 trial with 134 patients were released in January, and found that mean annualized bleed rate was reduced by 80% and Factor VIII usage was reduced by 94% in year three. At the end of year 3, 92% of patients remained off prophylaxis.

Roctavian was approved by European authorities in August 2022.

Another expected gene therapy is beremagene geperpavec (B-VEC) to treat patients with dystrophic epidermolysis bullosa (DEB), an incurable and often fatal skin blistering condition. It is caused by a lack of collagen protein in the skin and results from mutations in the COL7A1 gene encoding type VII collagen.

Developed by Krystal Biotech, B-VEC delivers functional human COL7A1 genes directly to the skin of affected patients. In January, the FDA extended by three months the PDUFA date to May 19, 2023 (originally Feb. 17, 2023). The extension was related to the submission of additional manufacturing information that will require additional time to review. Data from a phase 3 trial of 31 patients were published in the New England Journal of Medicine. Complete wound healing at six months occurred in 67.4% of B-VEC wounds compared with 21.6% for placebo.

A third expected gene therapy is delandistrogene moxeparvovec, developed by Sarepta Therapeutics to treat patients with Duchenne muscular dystrophy, has a PDUFA date of May 29, 2023. Duchenne is characterized by a mutation in the dystrophin gene that results in the lack of dystrophin, which acts as a shock absorber for muscle at the membrane. Delandistrogene moxeparvovec delivers to muscle a gene that codes for a shortened, functional form of dystrophin.

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