This quarterly review of new drug applications contains data for applications approved for the first time during the fourth quarter of 2021, which includes New Molecular Entities (NMEs) and new biologics. A total of 40 applications were approved by the FDA during these three months. The following are the different percentages based on the type of application – Type I (New molecular entity, 15% small molecules, 10% BLA), Type 2 (New active ingredient, such as a salt, 5%), Type 3 (New dosage form, 25%), Type 4 (New combination, 5%), Type 5 (New formulation or manufacturer, 30%), Gas (2.5%) and BLA (Biologics license application, 17.5%).
Approvals in the last quarter of 2021 showed five main routes of administration – Injection (47.5%), Oral (40%), Nasal (7.5%), Ophthalmic (2.5%) and Vaginal (2.5%). For oral routes, tablets (22.5%) and capsules (10%) were most widely used, but one application each used granules in a pouch, oral suspension and oral solution formulations. Medical Carbon Dioxide USP was approved for Middlesex Gases Technologies.
Medical Carbon Dioxide is used for various medical purposes. It can be used as an insufflation gas for minimal invasive surgery (laparoscopy, endoscopy, athroscopy) to enlarge and stabilize body cavities for better visibility of the surgical field. Also, in its liquid phase, it can be used to provide temperatures down to - 76° C for cryotherapy or for local analgesia by external application onto the skin surface.
Scemblix (Asciminib HCl) received orphan drug status, was given fast track approval, and was designated as a breakthrough therapy. Scemblix belongs to a class of drugs called Tyrosine Kinase Inhibitors. It is used to treat the symptoms of Chronic Myeloid Leukemia.
Voxzogo (Vosoritide), a first-in-class drug, obtained orphan drug designation and received priority/accelerated review. Voxzogo is used to increase linear growth in children with achondroplasia who are 5 years of age and older with open growth plates (epiphyses).
Fyarro (sirolimus protein-bound particles for injectable suspension), is a new formulation of sirolimus. It is an inhibitor of mechanistic target of rapamycin kinase (mTOR). It was approved to treat locally advanced unresectable or metastatic perivascular epithelioid cell tumor (PEComa). The formulation is provided as a lyophilized powder, which is diluted with 0.9% sodium chloride. Sodium chloride needs to be added slowly in the vial as it produces foam. If the medical device contains silicone oil as a lubricant, the product may form proteinaceous strands.
Cytalux (Pafolacianine sodium) is another first-in-class drug with orphan drug designation. It is an imaging agent and it helps identify ovarian cancer lesions during surgery. Ovarian cancer is hard to detect early, making it difficult to successfully treat.