2023 FDA Approvals Spotlight: Nearly 70 Novel Drugs Including 20 for Rare Diseases

　　【Pharmaceutical Network Industry Dynamics】The United States defines rare diseases as those with fewer than 200,000 patients per year (or a prevalence rate of less than 1 in 1,500). To address the situation where these patients have no available treatments, pharmaceutical companies have made significant efforts in recent years. 2023 can be considered a fruitful year for advancements in the treatment of rare diseases. According to industry statistics, the FDA approved nearly 70 new drugs in 2023, 20 of which were for the treatment of rare diseases, offering new therapeutic options for many patients with rare conditions.
 

Many new drugs for rare diseases are noteworthy. For example, in February 2023, Lamzede (velmanase alfa-tycv), a treatment for α-Mannosidosis (AM), received FDA approval for the treatment of non-central nervous system manifestations of α-Mannosidosis (AM) in adult and pediatric patients. It is reported that α-Mannosidosis (AM) is a disease caused by genetic variations in the MAN2B1 gene, which encodes lysosomal α-mannosidase, and there is a significant unmet treatment need for patients. According to FDA data, one in every 500,000 people is affected by this disease.
 

Fabry disease is a lysosomal storage disorder caused by mutations in the gene encoding α-galactosidase, leading to insufficient activity of the enzyme α-galactosidase A, which is essential for the metabolism of globotriaosylceramide (Gb3). The current standard treatment for Fabry disease is enzyme replacement therapy. On May 11, 2023, the U.S. FDA approved a pegylated enzyme replacement therapy (ERT) called Elfabrio (pegunigalsidase alfa) for the treatment of adult patients with Fabry disease.
 

Hereditary ataxia (HA) is a group of neurodegenerative genetic disorders characterized primarily by ataxia as the main clinical manifestation. Developing effective drugs for this condition is urgently needed. On March 1, 2023, Reata Pharmaceuticals announced that the FDA had approved its novel drug omaveloxolone (brand name SKYCLARYS™) for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older. Additionally, with this approval, the company received a Rare Pediatric Disease Priority Review Voucher (PRV) from the FDA.
 

Rett Syndrome is a rare genetic neurologic and developmental disorder that affects the development of the brain. Patients with Rett syndrome gradually lose motor skills and language abilities. On March 10, 2023, the FDA approved trofinetide oral solution (Daybue) as the first treatment for Rett syndrome, indicated for adults and children aged 2 years and older with Rett syndrome. It is reported that patients can take it orally or via a gastrostomy tube. The dosage is determined based on the patient's weight. It is taken twice daily, in the morning and evening, without being affected by food intake.
 

For instance, according to an announcement by GSK on September 15, 2023, the U.S. FDA has approved Ojjaara (momelotinib) for the treatment of intermediate or high-risk myelofibrosis in adult patients with anemia, including primary myelofibrosis or secondary myelofibrosis (post-polycythemia vera and post-essential thrombocythemia). According to GSK, about 40% of patients with myelofibrosis have moderate to severe anemia at diagnosis, and nearly all will develop anemia during the course of the disease. This condition is associated with Janus kinase (JAK) proteins, the dysregulation of which plays a role in inflammation and certain cancers.
 

In addition to the new drugs in the field of rare disease treatment mentioned above, the new drug indications for rare diseases approved by the FDA in 2023 also cover hot flashes caused by menopause, late-onset Pompe disease in adults, epidermolysis bullosa, and primary hyperoxaluria type 1, which will bring good news to patients with related rare diseases.
 

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