In a report published this morning, the Personalized Medicine Coalition (PMC) shows that personalized medicines topped one third of new U.S. Food and Drug Administration (FDA) drug approvals for the fourth year in a row in 2023.

The trend toward personalized treatment approvals is especially pronounced in the rare disease space, where the number of new treatment approvals more than doubled last year. In 2023, FDA approved 16 new personalized treatments for rare disease patients, up from six in 2022. The newly approved personalized treatments for 2023 also include seven cancer drugs and three for other diseases and conditions.

The annual report, titled Personalized Medicine at FDA: The Scope & Significance of Progress in 2023, documents the progress of companies in the diagnostics and biopharmaceutical industry that are working to bring to market new tests and treatments that will help make medicine more efficient and effective by targeting the right treatments to the right patients at the right times. The report explores the significance of 26 new personalized treatment approvals, 19 expanded indications for existing personalized medicines, and 17 significant new or expanded indications for 12 diagnostic testing products.

“The tests and treatments spotlighted in Personalized Medicine at FDA: The Scope & Significance of Progress in 2023, if they are made widely accessible, can go a long way to make health care more efficient and effective for patients with rare diseases, cancers, and other conditions,” said PMC President Edward Abrahams.

26 Newly Approved Personalized Treatments — Including 20 New Molecular Entities and Six Newly Approved Gene/Cell-Based Therapies

Rare Diseases

1. Casgevy (exagamglogene autotemcel) — for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso occlusive crises.
2. Elevidys (delandistrogene moxeparvovec-rokl) — for the treatment of ambulatory pediatric patients with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.
3. Elfabrio (pegunigalsidase alfa-iwxj) — for the treatment of Fabry disease.
4. Fabhalta (iptacopan) — for the treatment of paroxysmal nocturnal hemoglobinuria.
5. Filspari (sparsentan) — for the treatment of proteinuria associated with primary immunoglobulin A nephropathy in patients at risk of rapid disease progression.
6. Joenja (leniolisib) — for the treatment of activated phosphoinositide 3-kinase delta syndrome.
7. Lamzede (velmanase alfa-tycv) — for the treatment of non-central nervous system manifestations of alpha-mannosidosis.
8. Lyfgenia (lovotibeglogene autotemcel) — for the treatment of patients 12 years of age or older with sickle cell disease and a history of vaso occlusive events.
9. Pombiliti (cipaglucosidase alfa-atga) — for the treatment of Pompe disease.
10. Qalsody (tofersen) — for the treatment of amyotrophic lateral sclerosis.
11. Rivfloza (nedosiran) — for the treatment of high urinary oxalate levels in patients with primary hyperoxaluria type 1.
12. Roctavian (valoctocogene roxaparvovec-rvox) — for the treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity <1 IU/dL) without pre-existing antibodies to adeno-associated virus serotype 5.
13. Sohonos (palovarotene) — for the reduction in volume of new heterotopic ossification in patients with fibrodysplasia ossificans progressiva.
14. Veopoz (pozelimab-bbfg) — for the treatment of CHAPLE disease.
15. Vyjuvek (beremagene geperpavec-svdt) — for the treatment of wounds in patients with dystrophic epidermolysis bullosa with mutation(s) in the collagen type VII alpha 1 chain gene.
16. Wainua (eplontersen) — for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis.

Cancers

1. Augtyro (repotrectinib) — for the treatment of metastatic non-small cell lung cancer.
2. Loqtorzi (toripalimab-tpzi) — for the treatment of metastatic advanced nasopharyngeal carcinoma.
3. Omisirge (omidubicel-onlv) — for use in patients with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning to reduce the time to neutrophil recovery and the incidence of infection.
4. Orserdu (elacestrant) — for the treatment of metastatic breast cancer.
5. Truqap (capivasertib) — for the treatment, in combination with fulvestrant, of metastatic breast cancer.
6. Vanflyta (quizartinib) — for the treatment, in combination with cytarabine and anthracycline, of newly diagnosed acute myeloid leukemia following consolidation chemotherapy.
7. Zynyz (retifanlimab-dlwr) — for the treatment of metastatic Merkel cell carcinoma.

Other Diseases

1. Leqembi (lecanemab-irmb) — for the treatment of Alzheimer’s disease.
2. Rystiggio (rozanolixizumab-noli) — for the treatment of generalized myasthenia gravis.
3. Zilbrysq (zilucoplan) — for the treatment of generalized myasthenia gravis.

When evaluating new molecular entities, PMC categorizes personalized medicines as those therapeutic products for which the label includes reference to specific biological markers, often identified by diagnostic tools, that help guide decisions and/or procedures for their use in individual patients.