The regulator has approved a treatment for beta thalassemia and sickle cell disease, making the UK the first country to approve a therapeutic based on Crispr gene editing.
The treatment known as Casgevy, created by Crispr Therapeutics and Vertex Pharmaceuticals, has received approval from the Medicines and Healthcare Products Regulatory Agency. The medicine may take the place of bone marrow transplants.
As it gets permission to reduce its workload by adopting other country’s recommendations for drug approvals starting next year, the UK regulator has pledged to concentrate on bringing the most innovative therapies to market as quickly as possible. Following the UK’s exit from the EU, where it had played a significant role in the bloc’s regulatory agency, it had been finding it difficult to stay up due to resource constraints.
In under 11 years, Crispr—a flexible and efficient gene editing tool—went from its original discovery to an approved medication. It is based on the bacterial immune system. After demonstrating in 2012 that Crispr might be used to disrupt, erase, or fix genomic mistakes, scientists Jennifer Doudna and Emmanuelle Charpentier were awarded a Nobel Prize in 2020.
Though the UK accounts for little more than 2 percent of the world drug market—a significantly smaller share than it did when it was a member of the EU—the MHRA is aiming to draw producers of novel treatments to the country.
An mRNA vaccination for Covid-19 was approved by UK regulators first worldwide. However, it has come under fire after the epidemic for lagging behind and creating delays for commercial clinical trials. It has now caught up and brought its trial approval schedules back to comply with the law.
The agency was granted an additional £10 million in funding over a two-year period by UK chancellor Jeremy Hunt in the March Budget with the goal of “put in place the quickest, simplest, regulatory approval in the world for companies seeking rapid market access.”
Sickle cell disease and beta thalassemia, according to Julian Beach, interim executive director of healthcare quality and access at the MHRA, are “painful, life-long conditions that in some cases can be fatal.” In the UK, sickle cell disease affects about 15,000.
“To date, a bone marrow transplant—which must come from a closely matched donor and carries a risk of rejection—has been the only permanent treatment option,” he added.
Casgevy is a difficult medicine to take. To modify a patient’s genes in a lab, stem cells from the patient’s bone marrow must be removed. Patients must stay in the hospital for at least a month after the altered cells are allowed to return into their bodies before their bodies start to produce regular red blood cells.
Because it is simpler to harvest the cells and alter their genes, Crispr has been used initially to treat blood diseases. To address other severe genetic conditions such as those affecting the liver or eyes, other businesses are developing methods to alter cells inside the body.