Only a couple of years since the first sickle cell disease (SCD) gene therapies gained U.S. FDA approval, researchers are working to expand access for younger children, and to improve manufacturing and commercialization to reach patients faster.
Industry watchers were surprised when Eli Lilly and Co.’s oral GLP-1 candidate, orforglipron, did not appear among the first nine recipients of the U.S. FDA’s commissioner’s national priority voucher (CNPV) program aimed at shortening regulatory review times, boosting domestic manufacturing and improving affordability. But orforglipron, which recently nailed endpoints in a second phase III trial and has been hailed a potentially best-in-class compound, was among the second batch of six drugs added to the CNPV list.
A total of 33 states, plus the District of Columbia and Puerto Rico, agreed to participate in the U.S. CMS’ voluntary, outcomes-based program aimed at helping state Medicaid programs cover high-priced cell and gene therapies, starting with therapies for sickle cell disease.
The U.S. CMS has negotiated outcomes-based agreements with Bluebird Bio Inc. and Vertex Pharmaceuticals Inc. to make their costly sickle cell gene therapies the first treatments to become available through the voluntary Medicaid Cell and Gene Therapy Access Model.
Alongside the release of abstracts related to the American Society of Hematology meeting next month in San Diego, and as part of the firm’s third-quarter update, Beam Therapeutics Inc. disclosed that one patient died in the phase I/II trial testing BEAM-101 in sickle cell disease (SCD).
Although more and more gene therapies are getting the FDA stamp of approval, concerns persist about their potential long-term risks. U.S. lawmakers have proposed several pieces of legislation over the past few years to address some of the uncertainties. Now the Congressional Research Service (CRS) is suggesting other requirements Congress may want to consider to improve the regulatory landscape for gene therapies, especially those intended to treat blood disorders.
In denying Medicaid patients with sickle cell disease or transfusion-dependent beta-thalassemia access to Vertex Pharmaceuticals Inc.’s fertility preservation program, which is intended to counteract a side effect of the company’s gene-editing therapy, Casgevy, “the federal government now stands as the barrier between thousands of predominantly Black Americans and the necessary medical care that would protect their basic right to have biological children,” Vertex said in a lawsuit filed July 15.
Orum Therapeutics Inc. struck a potential $945 million (₩1.3 trillion) deal with Vertex Pharmaceuticals Inc. to discover novel degrader antibody conjugates (DAC) as targeted conditioning agents for use with gene editing, including Vertex’s gene therapy, Casgevy (exagamglogene autotemcel).
Some gene therapies could be big winners under the changes the U.S. Centers for Medicare & Medicaid Services (CMS) is proposing to Medicare’s new technology add-on program (NTAP) for its fiscal 2025 inpatient prospective payment system.
The European Commission approved two therapies for progressive, genetic diseases: Biogen Inc.’s Friedreich’s ataxia drug, Skyclarys (omaveloxolone), and Crispr Therapeutics AG’s CRISPR/Cas9 gene therapy for sickle cell disease and transfusion-dependent beta-thalassemia, Casgevy (exagamglogene autotemcel, exa-cel).
