New Drugs Approved by FDA in February 2022

1. Ruzurgi

The active ingredient of "Ruzurgi" (amifampridine) is diaminopyridine, which is indicated for the treatment of Lambert-Eaton syndrome myasthenia. The dosage form is tablet, which is a small molecule drug.

The drug was approved by the FDA in 2019, but was immediately sued by the US company CATALYST to the FDA, alleging that the drug's active ingredient violated another trade name "Firdapse" (an FDA-approved treatment for Lambert in 2018) -Eaton syndrome peripheral neuropathy drug) exclusive rights to the drug, after the court changed "Ruzurgi" to provisional approval.

2. Fleqsuvy

The active ingredient "Fleqsuvy" is baclofen, which is in the form of an oral suspension, a gamma-aminobutyric acid (GABA) agonist for the treatment of multiple sclerosis (MS) or spinal cord injury and other spinal cord diseases caused by Spasticity, especially relief of flexor spasm associated with pain, clonus, and muscle stiffness, is not indicated for the treatment of skeletal muscle spasm caused by rheumatic diseases.

3. Enjaymo

"Enjaymo" (sutimlimab) is the first FDA-approved monoclonal antibody for the treatment of cold agglutinin disease. It is in the form of injection and reduces the need for red blood cell (RBC) transfusion in patients due to hemolysis by inhibiting the destruction (hemolysis) of red blood cells.

The approval was based on results from a Phase III clinical trial: Enjaymo met the primary efficacy endpoint, with 54% of patients meeting the primary composite endpoint. The product has received orphan drug, breakthrough therapy and priority reviewqualifications, and is currently in new drug applications in Japan and Europe.

4. Vonjo

"Vonjo" (pacritinib) is a novel oral JAK kinase inhibitor indicated for the treatment of patients with myelofibrosis with severe thrombocytopenia. Myelofibrosis is a myeloproliferative disorder in which fibrous scar tissue forms in patients, leading to severe thrombocytopenia and anemia. The drug is a kinase inhibitor and does not inhibit JAK1, but specifically inhibits JAK2 and JAK1. Previously, Vonjo has been granted priority review status by the FDA.

5. Carvykti

"Carvykti" (Sidaki Olenza) is a BCMA-targeted CAR-T cell therapy for the treatment of patients with relapsed or refractory multiple myeloma (R/R MM) who have received four prior line or above, including proteasome inhibitors, immunosuppressants, and anti-CD38 monoclonal antibodies.

This drug is Legend Bio's first CAR-T cell therapy, which is of great significance. It was granted orphan drug designation by the US FDA and European EMA in February 2019 and February 2020, respectively.