In 2023, the Centers for Drug Evaluation and Research of FDA in the US approved a total of 55 new drugs, of which 24% were targeted at rare diseases, which is a historic high. Patients with hereditary immunodeficiency diseases are offered new treatment options, for example:

​

1. The anti β Amyloid protein monoclonal antibody Leqembi or lecanemab, jointly launched by Biogen and Eisai, is the first FDA approved new drug for Alzheimer's disease in 20 years.

2. Jaypirca from Eli Lilly is the first non covalent BTK inhibitor used for mantle cell lymphoma.

3. Skyclaims (omaveloxolone) from Reata Pharmaceuticals is the first drug to treat Friedreich's Ataxia, or FA.

4. Biogen and Ionis jointly launched the antisense oligonucleotide therapy Qalsody (tofersen), which is the first gene targeted therapy for the treatment of amyotrophic lateral sclerosis, or ALS.

5. Seres Therapeutics' oral microbiota therapy Vowst, SER-109, is the world's first oral fecal microbiota therapy used to prevent recurrent Clostridium difficile infections.

6. GSK's respiratory syncytial virus (RSV) vaccine Arexvy is the world's first RSV vaccine used to prevent lower respiratory tract diseases caused by RSV infection in people aged 60 and above.

7. BioMarin's gene therapy Roctavian is the first gene therapy for type A hemophilia.

8. Johnson&Johnson's Talvey (talquetamab) is the world's first GPRC5D/CD3 dual antibody for multiple myeloma.

9. Novartis Fabhalta (iptacopan) is the first oral monotherapy for adult paroxysmal nocturnal hemoglobinuria, or PNH.

10. The CRISPR/Cas9 gene editing therapy, Casgevy, jointly developed by Vertex and CRISPR, is the first CRISPR gene editing drug for sickle cell disease.