Approval for Lamzede

On Feb. 16, 2023, Chiesi Global Rare Diseases earned approval for Lamzede^® (velmanase alfa-tycv) injection from the U.S. Food and Drug Administration (FDA). It treats the non-central nervous system (CNS) symptoms of an extremely rare progressive disease, alpha-mannosidosis. Caused by mutated MAN2B1 genes, the hereditary condition results in the deficiency of an enzyme that metabolizes some complex sugars. Its symptoms include a large head and forehead, enlarged gums and tongue, frequent infections, cognitive impairment, and problems with hearing, moving, and speaking. Only about one or two people per one million are believed to have the disorder. Current treatment of alpha-mannosidosis focuses on relieving symptoms with numerous specialty physicians and other healthcare providers typically involved in therapy. Stem cell transplants have been successful for some patients. Lamzede is the first drug to address the cause of alpha-mannosidosis. Replacing the missing enzyme increases the breakdown of complex sugars, reduces their accumulation in the body, and decreases organ damage. Indicated for intravenous (IV) infusions once every week at 1mg/kg, it will be packaged in single-dose vials containing 10mg of powder for reconstitution. Its labeling has a boxed warning that cautions against hypersensitivity reactions among some patients receiving it, so injections should be given in a facility that is staffed and equipped to handle severe reactions. Patients using it also have experienced fever, headaches, joint pain, and upper respiratory infections. Before infusions, patients may need to have pretreatment with an antihistamine, a corticosteroid, and/or a fever-reducing agent. Plans for launching, distributing, and pricing Lamzede have not been made public. Here is its complete prescribing information.

Cardiovascular Indication for Synjardy/XR

The FDA approved a new indication on Feb. 6, 2023, for Boehringer Ingelheim’s empagliflozin and metformin fixed-dose combinations, Synjardy^® tablets, and Synjardy® XR extended-release tablets. Containing a sodium-glucose co-transporter 2 (SGLT2) inhibitor and a biguanide, both have been approved for several years to treat adults who have type 2 diabetes. They now can be used to lower the risk of death due to cardiovascular (CV) causes for adults who have CV disease and to decrease the chances of death or hospitalization due to heart failure (HF) for adults who have HF. Some other single-ingredient SGLT 2 inhibitors have similar CV indications whether or not the patient has diabetes, but — due to their metformin components — Synjardy and Synjardy XR should not be used for patients who do not have diabetes. All products that contain metformin have a boxed warning about increased chances of lactic acidosis, a potentially dangerous accumulation of acid in the blood. Older adults, patients who have kidney conditions, and those who have liver disorders may be at higher risk. Synjardy is taken twice a day; Synjardy XR is once daily. Revised prescribing information is here for Synjardy and here for Synjardy XR.

Cibinqo Approved for Adolescents

Cibinqo^® (abrocitinib – Pfizer) tablets is an oral Janus-associated kinase (JAK) inhibitor. It originally was FDA approved in January 2022 for treating adults who have moderate-to-severe atopic dermatitis that has not responded to treatment or that has recurred after therapy with other systemic drugs. On Feb. 9, 2023, the FDA extended the indication to include patients between the ages of 12 years old and 18 years old. The recommended dose for all patients is 100mg once a day. Cibinqo should not be taken if the patient is being treated with any other immunosuppressant, a biologic immunomodulator, or another JAK inhibitor. Labeling for Cibinqo carries the same boxed warnings as all oral drugs in the class that are used to treat inflammatory conditions. They summarize possibly increased risks of blood clots, cancer, and serious infections that may be associated with the use of a JAK inhibitor. Patients taking or using one of them also should be screened for tuberculosis (TB) before starting treatment and periodically while therapy continues. For Cibinqo’s current prescribing information, look here.