Note: This story was updated Dec. 12, 2023, to clarify that Lyfgenia treats vaso-occlusive events, including painful vaso-occlusive crises (VOCs). The U.S. Food and Drug Administration (FDA) has approved the gene-editing therapy exagamglogene autotemcel (exa-cel) — now known as Casgevy — for treating sickle cell disease (SCD). The therapy…

Note: This story was updated Dec. 12, 2023, to clarify that Lyfgenia treats vaso-occlusive events (VOEs), including painful vaso-occlusive crises (VOCs). The U.S. Food and Drug Administration (FDA) has approved lovotibeglogene autotemcel, a gene therapy from Bluebird Bio known as lovo-cel, for treating sickle cell disease (SCD) patients ages…

The U.S. Food and Drug Administration (FDA) has placed a clinical hold on FTX-6058, an experimental therapy that Fulcrum Therapeutics is developing to treat sickle cell disease (SCD). Fulcrum announced it would be stopping dosing in an ongoing Phase 1b clinical trial (NCT05169580) that’s testing different…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

By 2029, Americans with sickle cell disease (SCD) could be living 10 years longer than they do today, predicts U.S. Health and Human Services Secretary Alex Azar. That’s one of the Trump administration’s long-term goals, Azar — the nation’s top health official — told more than 900 delegates attending…

Siklos (hydroxyurea 100 mg tablets) — indicated for the treatment of pediatric patients, ages 2 and older, with sickle cell anemia (SCA) — can now be ordered in U.S. pharmacies. Developed by French pharmaceutical company ADDMEDICA, Siklos is now exclusively marketed in the U.S. by Medunik USA…

The U.S. Food and Drug Administration (FDA) approved Imbruvica (ibrutinib) for treatment of adult patients with chronic graft-versus-host-disease (cGVHD) who failed to respond to other forms of systemic therapy, AbbVie announced. FDA’s decision means Imbruvica is the first and only therapy specifically approved for adults with cGVHD, a serious outcome that…