Slideshow: 2023 Biologic Product Approvals

Biologic product approvals in 2023¹ ran the gamut, including 2 new therapies to treat hemophilia A, a gene therapy for pediatric Duchenne muscular dystrophy, and the first orally administered fecal microbiota product to treat recurrent C difficile infection. And on December 8, the FDA approved 2 new therapies—the first of their kind—to treat sickle cell disease, an inherited blood disorder that disproportionately affects Black or African American individuals.

References

1. 2023 biological license application approvals. FDA. Reviewed November 27, 2023. Accessed November 28, 2023. https://www.fda.gov/vaccines-blood-biologics/development-approval-process-cber/2023-biological-license-application-approvals

2. FDA approves once-weekly Altuviiio, a new class of factor VIII therapy for hemophiia A that offers significant protection. News release. Sanofi. February 23, 2023. Accessed November 28, 2023. https://www.sanofi.com/en/media-room/press-releases/2023/2023-02-23-21-00-00-2614759

3. FDA approves cell therapy for patients with blood cancers to reduce risk of infection following stem cell transplantation. News release. FDA. April 17, 2023. Accessed November 28, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-cell-therapy-patients-blood-cancers-reduce-risk-infection-following-stem-cell

4. FDA approves first orally administered fecal microbiota product for the prevention of recurrence of Clostridioides difficile infection. News release. FDA. April 26, 2023. Accessed November 28, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-orally-administered-fecal-microbiota-product-prevention-recurrence-clostridioides

5. FDA approves first topical gene therapy for treatment of wounds in patients with dystrophic epidermolysis bullosa. News release. FDA. May 19, 2023. Accessed November 28, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-topical-gene-therapy-treatment-wounds-patients-dystrophic-epidermolysis-bullosa

6. FDA approves first gene therapy for treatment of certain patients with Duchenne muscular dystrophy. News release. FDA. June 22, 2023. Accessed November 28, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-certain-patients-duchenne-muscular-dystrophy

7. Sarepta Therapeutics announces topline results from EMBARK, a global pivotal study of Elevidys gene therapy for Duchenne muscular dystrophy. News release. Sarepta Therapeutics. October 30, 2023. Accessed November 28, 2023. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-topline-results-embark-global-0

8. FDA approves first cellular therapy to treat patients with type 1 diabetes. News release. FDA. June 28, 2023. Accessed November 28, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-cellular-therapy-treat-patients-type-1-diabetes

9. FDA approves first gene therapy for adults with severe hemophilia A. News release. FDA. June 29, 2023. Accessed November 28, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-adults-severe-hemophilia

10. Octapharma’s prothrombin complex concentrate, Balfaxar, receives FDA approval for warfarin reversal in urgent surgery and invasive procedures. News release. Octapharma. July 27, 2023. Accessed November 28, 2023. https://www.octapharma.com/news/press-release/2023/octapharma-s-prothrombin-complex-concentrate-balfaxar-receives-fda-approval-for-warfarin-reversal-in-urgent-surgery-invasive-procedures

11. Vertex and CRISPR Therapeutics announce US approval of Casgevy (exagamglogene autotemcel) for the treatment of sickle cell disease. News release. Vertex. December 8, 2023. Accessed December 18, 2023. https://news.vrtx.com/news-releases/news-release-details/vertex-and-crispr-therapeutics-announce-us-fda-approval

12. FDA approves first gene therapies to treat patients with sickle cell disease. News release. FDA. December 8, 2023. Accessed December 18, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease

13. Bluebird bio announces FDA approval of Lyfgenia (lovotibeglogene autotemcel) for patients ages 12 and older with sickle cell disease and a history of vaso-occlusive events. News release. bluebird bio. December 8, 2023. Accessed December 18, 2023. https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-announces-fda-approval-lyfgeniatm-lovotibeglogene