The prospect of genetic engineering using CRISPR (clustered regularly interspaced short palindromic repeats) and CRISPR-associated nucleases (Cas) has long been hailed as a “revolutionary” development in medicine.

This technology is rapidly advancing, and several CRISPR/Cas-based drugs have entered clinical trials over the past several years. One kind of product in clinical trials is CRISPR-modified cells, such as CTX001 (CRISPR-Cas9-modified autologous hematopoietic stem cells), currently under study for the treatment of b-thalassemia and severe sickle cell anemia. Another CRISPR-based product, AGN-151587, is injected into the eye with the goal of eliminating a genetic mutation in patients with Leber congenital amaurosis 10, a leading cause of childhood blindness. In parallel, others are working to harness the CRISPR/Cas system to develop drugs for rare diseases, including bespoke therapies tailored to an individual patient’s needs.

“Orphan” drug exclusivity, which is intended to reward drug companies’ investment in the development of certain drugs, might soon be harder to get—and keep.

Over the past several months, Congress introduced two similar bills to amend a “loophole” in the Orphan Drug Act (ODA).  On October 17, 2019, a bipartisan group of House members introduced H.R. 4712 (“Fairness in Orphan Drug Exclusivity Act”) (“the House bill”).  On February 11, 2020, bipartisan Senators sponsored a companion bill bearing the same title (S. 3271) (“the Senate bill”).  Consistent with recent political interest in curbing high drug prices, the proposed legislation is intended to limit the availability of orphan drug exclusivity for certain drugs, with the goal of thereby promoting competition.