What happened
Shares of CRISPR Therapeutics (CRSP 3.22%) were up 15% Thursday morning, a day after a drug-pricing group said the company's single-dose therapy for sickle cell disease (SCD) could be cost effective if priced below $1.9 million.
So what
CRISPR is developing exa-cel, a gene-editing therapy, with Vertex Pharmaceuticals. The companies are awaiting word from the Food and Drug Administration (FDA) if exa-cel will be approved to treat SCD and transfusion-dependent beta thalassemia (TDT), two rare genetic blood disorders.
The Institute for Clinical and Economic Review (ICER) released a draft that said a price range under $1.9 million for exa-cel and lovo-cel (another gene-editing SCD therapy developed by Bluebird Bio) would be cost-effective compared to the costs currently associated with the long-term treatment of the disease. ICER is not a government agency and this is just a draft report, so the findings aren't binding, but they do hold some weight with insurance companies.
"Sickle cell disease can affect nearly every organ system in the body, and severe sickle cell disease affects nearly every aspect of a person's life," ICER's Chief Medical Officer, David Rind, said in a release. "From the earliest days of gene therapy, patients, families, and clinicians have imagined that someday it might be possible to address the underlying genetics of sickle cell to achieve a cure. These first two genetic therapies, using different technologies and altering different genetic targets may mean that day has nearly arrived."
Now what
The key for CRISPR, and Vertex, is whether the therapy is approved by the FDA. CRISPR is a clinical-stage biotech and its shares are down more than 32% over the past year. The companies are expected to hear in the first half of this year from the FDA and if the drug is approved, the stock will likely jump even higher.
The companies said they completed the rolling submission for the therapy's biologics license applications for SCD and TDT on April 3. The therapy has already been given Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA for SCD and TDT.