NICE not sold on Vertex, CRISPR's groundbreaking sickle cell gene therapy Casgevy without more data

Ever since Vertex and CRISPR therapeutics scored a groundbreaking U.K. authorization for their sickle cell gene therapy Casgevy in November, the looming hurdle to commercialization was securing a recommendation from the region’s cost watchdog.

Now, the National Institute for Health and Care Excellence (NICE), a drug pricing gatekeeper for the nationally-run health service in England, has revealed that it’s not quite sold on the med.

In its draft guidance (PDF), NICE did not recommend Casgevy but is “exploring further data collection” on the therapy’s effectiveness and a potential commercial arrangement, the regulator said in an emailed press release.

As it stands, the only curative treatment available in the U.K. for patients with severe sickle cell disease with recurrent vaso-occlusive crises is a stem cell transplant from a matching donor.

Considering the “small numbers” of patients that can get such a treatment, the NICE committee as well as clinical and patient experts agreed that “it is possible” that Casgevy could offer a cure to a larger population, it noted.

But to make its final ruling, the regulator will need Vertex to consider additional data collection beyond its current proposal for access, which could stand to “address some additional clinical uncertainties.” NICE is asking the company for an updated managed access proposal in response to its draft guidance.

Casgevy, which is also known as exa-cel, made history with its nod from the U.K.’s Medicines and Healthcare products Regulatory Agency in November. Vertex said at the time that the authorization could open up the treatment to 2,000 U.K. patients.

The drug’s steep price and Vertex’s history with NICE, however, pointed to potential troubles ahead in getting the therapy to England’s National Health Services (NHS).

Earlier in November, NICE released a draft guidance on the company’s cystic fibrosis drugs cocktails Trikafta (which is branded as Kaftrio in the U.K.), Symkevi and Orkambi, recognizing the effectiveness of the therapies but stopping short of labeling them as cost-effective.  

Meanwhile, the FDA approved Casgevy in December following a positive advisory committee vote in October. The company said in an SEC filing that it will charge $2.2 million for the therapy.

The U.S.’s Institute for Clinical and Economic Review (ICER) had previously put cost-effectiveness for Casgevy and bluebird bio’s Lyfgenia gene therapy at a $1.35 million to $2.05 million range.

More recently, the agency approved the same drug to treat transfusion-dependent beta thalassemia (TDT).