FDA Approved Four New Gene Therapies, Other Novel Agents in 2022

· 10 min read

While the FDA may not have approved the most drugs in a year in 2022, it still gave the green light to a number of agents, many of them specialty medications, as well as granted additional indications to existing therapies. The FDA’s Center for Drug Evaluation and Research (CDER) approved 39 new molecular entities in 2022, and the Center for Biologics Evaluation and Research (CBER) approved 12 biologic license applications, including four new gene therapies in the second half of the year. AIS Health, a division of MMIT, asked some industry sources what the most notable 2022 FDA approvals were and why they were so important.

Arash Sadeghi, Pharm.D., clinical pharmacist at Optum Rx, Inc.: Prior to 2022, we had two true gene therapies approved by the FDA — Luxturna and Zolgensma. But in 2022 alone, there were three additional gene therapies approved, including Skysona (elivaldogene autotemcel) [from bluebird bio, Inc.] for cerebral adrenoleukodystrophy, Zynteglo (betibeglogene autotemcel) [from bluebird] for beta-thalassemia and [CSL Behring LLC’s] Hemgenix (etranacogene dezaparvovec-drlb) for hemophilia B. [Ferring Pharmaceuticals’ Adstiladrin (nadofaragene firadenovec-vncg) was the fourth gene therapy to gain approval, which came after AIS Health’s deadline for comments.] The one-time cost for these gene therapies ranged from $2.8 million to $3.5 million. We expect the number of gene therapies to continue to grow in 2023.

Mounjaro (tirzepatide) [from Eli Lilly and Co.] was the first dual acting GIP/GLP-1 receptor agonist approved by the FDA for the treatment of type 2 diabetes. While many other drugs are available for type 2 diabetes, including other GLP-1 receptor agonists, this is a growing category because of the use of these drugs for chronic weight management, and that trend is potentially going to continue if tirzepatide is eventually approved for this use as well.

Tzield (teplizumab-mzwv) [from Provention Bio, Inc.] became the first treatment approved to delay the onset of clinical type 1 diabetes. The CDC estimates that the annual number of newly diagnosed cases of type 1 diabetes is 18,291 children and adolescents younger than 20 years of age. Tzield is administered as a 14-day treatment course and costs $193,900 for the full regimen.

Andy Szczotka, Pharm.D., chief pharmacy officer at AscellaHealth: There are three notable, high-impact gene therapies that were approved. The most recent gene therapy approval is CSL Behring’s Hemgenix for hemophilia B.…Currently, the treatment for hemophilia B is IV infusions of congenital factor IX (FIX), which costs approximately $500,00 to $700,00 annually. Hemgenix provides a single-dose IV infusion that delivers an adeno-associated virus vector-based gene therapy carrying the gene that encodes for FIX. The cost for this single-use therapy is approximately $3.5 million.

Another notable approval was Skysona, a gene therapy used to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy. CALD is a rare, genetic condition that primarily affects young males and is caused by a mutation in the ABCD1 gene, located on the X chromosome, which affects the production of adrenoleukodystrophy protein (ALDP). The mutation results in the toxic buildup of very long-chain fatty acids in the brain and spinal cord, and this accumulation leads to progressive destruction of myelin. Prior to this approval, the only other treatment was stem cell transplant, which is associated with serious complications and mortality that increases in patients without a matched sibling donor. The cost for this single-use therapy is approximately $3 million.

Another gene therapy approved in 2022 was Zynteglo, which is an autologous hematopoietic stem cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular RBC transfusions. This one-time gene therapy works by adding functional copies of a modified form of the β-globin gene into a patient’s own hematopoietic stem cells. Prior to this approval, the only other option for people with this condition was regular red blood cell transfusions and iron chelation. The cost for this therapy is approximately $2.3 million.

Other notable products approved by the FDA in 2022 include:

Tzield, a single-dose infusion that has been proven to delay the onset of stage 3 type 1 diabetes.

Relyvrio (sodium phenylbutyrate/taurursodiol) [from Amylyx Pharmaceuticals, Inc.], the third agent approved for treatment of amyotrophic lateral sclerosis (ALS) in adults. In clinical studies, there was improvement in quality of life metrics using an ALS rating instrument for monitoring the progression and severity of disability in ALS patients. It will likely be used in combination with other approved ALS therapies (i.e., riluzole and edaravone).

Mounjaro, a dual action agent (GIP receptor and GLP-1 receptor agonist) used in adults with type 2 diabetes mellitus. Based on the clinical trial comparisons to other GLP-1 agents, it does seem that Mounjaro outperformed Trulicity and Ozempic in terms of A1c lowering, weight loss and percentage of patients achieving A1c <7%. Mounjaro will likely provide another therapeutic option for diabetes treatment at a higher cost than the currently available GLP-1 agents, but it may also hold a potential future role in weight management.

Sotyktu (deucravacitinib) [from Bristol Myers Squibb], a tyrosine kinase 2 (TYK2) inhibitor indicated for the treatment of adults with moderate-to-severe plaque psoriasis. This will provide another oral option that will compete directly with Otezla. In the clinical trials, Sotyktu patients were more likely to achieve PASI 75 at week 16 than patients on placebo or Otezla. It is priced at a premium to Otezla.

Nicole Kjesbo, Pharm.D., clinical program development director at Prime Therapeutics LLC: Prior to 2022, the last gene therapy approved was in 2019. In 2022, there have been three gene therapies approved: Zynteglo for transfusion-dependent beta thalassemia from bluebird bio priced at $2.8 million, Skysona for cerebral adrenoleukodystrophy from bluebird bio priced at $3 million, and Hemgenix for hemophilia B from CSL Behring priced at $3.5 million.

This is just the beginning; there are more than 10 gene therapies that may reach the FDA for review in 2023, and the numbers grow from there. Up until now, the gene therapies have been approved in relatively small disease states, but there are more coming that will treat a greater number of people, including for hemophilia A, sickle cell anemia and Duchenne muscular dystrophy.

Payers, providers and manufacturers will need to continue exploring novel payment and reimbursement models to counteract the high one-time cost associated with these gene therapies. Spreading costs over time and value-based contracting, linking reimbursement, coverage or payment to the real-world performance of the gene therapy, are options that are being explored.

Mesfin Tegenu, CEO and chairman of RxParadigm, Inc.: Drugs that bring new treatment options for patients and are considered new molecular entities bring innovation and advancement in the health care industry but also may bring some of the most potential earnings once they hit the market. Such products treat diseases including diabetes, psoriasis and other rare diseases.

Drew Walk, CEO of Soleo Health: There were several new FDA approvals in 2022. Two notable approvals were…oral medications for the treatment of amyotrophic lateral sclerosis (ALS): Radicava ORS (edaravone) oral suspension by Mitsubishi Tanabe and Amylyx’s Relyvrio [(sodium phenylbutyrate and taurursodiol)]. Both medications provided a new treatment alternative from the intravenous formulation of Radicava for appropriate eligible patients.

Winston Wong, Pharm.D., president of W-Squared Group: Specific to the oncology arena, the research is continuing and becoming more sophisticated, focusing on novel medication for orphan/rare and advanced relapsing/recurrent cancers. We are seeing the focus on the development of more next-generation biotherapeutics, including the CAR-T [i.e., chimeric antigen receptor T-cell] and NK [i.e., natural killer cell] treatment options; antibody-drug conjugates; making more traditional chemotherapy more effective and targeted; and bispecific antibodies, creating a dual approach to fighting the cancer. In addition, we should be expecting to see more products approved where the indication is based upon a biomarker and is tissue agnostic, especially with the greater acceptance of NGS [i.e., next-generation sequencing] testing. This is one area that payers must get a handle on for management, as their traditional criteria for approval has always been cancer/tissue specific.

Contact Kjesbo through Jenine Anderson at jenine.anderson@primetherapeutics.com, Sadeghi via Jennifer Statham at jennifer.statham@optum.com, Szczotka through Caroline Chambers at cchambers@cpronline.com, Tegenu at Mesfin.Tegenu@rxparadigm.com, Walk via Susan Turkell at sturkell@pairelations.com and Wong at w2sqgroup@gmail.com.

By Angela Maas

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