For 2021, Cell, Gene Therapies Make Prime's “Drugs to Watch” List


Three cell and gene therapies were among the drugs included in a specialty drugs pipeline forecast at the Academy of Managed Care Pharmacy 2021 meeting.

On the last day of the Academy of Managed Care Pharmacy (AMCP) 2021 meeting, 2 researchers from Prime Therapeutics reviewed new specialty therapies that may reach the market this year, including biologics and cell and gene therapies.

Catherine Starner, PharmD, BCPS, senior principal health outcomes researcher, and Nicole Kjesbo, PharmD, BCPS, clinical program development director-pipeline, said the drugs in their forecast (except for the cell and gene therapies) have been submitted to the FDA and are expected to have a high impact based on per member per month (PMPM), given their internal claims data, which draw from commercial lines, Medicare, and Medicaid, depending on the proposed indication.

In cell and gene therapies, Prime starts observing the drugs before they are submitted to the FDA. For their presentation, they reviewed 3 candidates they thought had the highest likelihood of being submitted:

  • Eladocagene exuparvovec, for aromatic l-amino acid decarboxylase (AADC) deficiency
  • Micro-dystrophin, or SRP-9001, for Duchenne muscular dystrophy (DMD)
  • Etranacogene dezaparvovec, or AMT-061, for severe and moderately severe hemophilia

Eladocagene exuparvovec. This drug, from PTC Therapeutics, provides a working copy of the DDC gene. AADC deficiency is extremely rare, affecting about 100 patients in the United States; one report said the 1-time infusion, surgically injected via MRI, will cost $4 million. Infants born with AADC deficiency fail to develop and have shortened life expectancy.

Micro-dystrophin. This candidate is 1 of 60 in the DMD pipeline and 1 of 2 from Sarepta Therapeutics. The gene transfer therapy is given by a single intravenous infusion. DMD affects between 500 to 5000 males worldwide. Other companies in the space include Pfizer, Solid Biosciences, and Astellas.

Etranacogene dezaparvovec. Severe and moderately severe hemophilia B affects 4000 males in the United States. There are 66 candidates for this indication in the pipeline; this one is from uniQure. Current treatment options are limited to factor IX products. Genentech has a similar therapy in development (fidanacogene elaparvovec).

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