Latest Updates & News
FDA Approval on February 16, 2023: LAMZEDE Breakthrough for Alpha-Mannosidosis Treatment
What is LAMZEDE prescribed for? For the treatment of non-central nervous system manifestations of alpha-mannosidosis in adult and pediatric patients. What is the name of the drug and what does it do? LAMZEDE (generic name: velmanase alfa-tycv), pronouonced “lam-zed,” is a medicine made from human genes that helps treat the non-brain...
Introducing POMBILITI: A New FDA-Approved Treatment for Late-Onset Pompe Disease as of September 28, 2023
What is POMBILITI prescribed for? POMBILITI is prescribed for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥88 lbs and who are not improving on their current enzyme replacement therapy (ERT). What is the name of the drug and what does it do? POMBILITI, pronounced...
EXXUA: A Groundbreaking Step for Treating Major Depression Approved on September 22, 2023
What is EXXUA prescribed for? EXXUA is prescribed to treat major depressive disorder in adults. What is the name of the drug and what does it do? EXXUA, pronounced EKS-shoo-uh, (generic name: gepirone) is a new medication approved by the FDA for treating adults with major depressive disorder (MDD). This drug offers a new option...
OJJAARA: A New Treatment for Myelofibrosis Approved by the FDA on September 15, 2023
What is OJJAARA prescribed for? OJJAARA is prescribed to treat intermediate or high-risk myelofibrosis in adults with anemia. What is the name of the drug and what does it do? OJJAARA, pronounced oh-JAR-uh, (generic name: momelotinib) is a medication approved on September 15, 2023, for the treatment of adults with intermediate or high-risk myelofibrosis,...
APHEXDA: A New Frontier in Multiple Myeloma Treatment Approved by the FDA on September 8, 2023
What is APHEXDA prescribed for? APHEXDA is used in combination with filgrastim (G-CSF) to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma. What is the name of the drug and what does it do? APHEXDA , pronounced ah fex’ dah, (generic name: motixafortide) is a...
FDA Approves VEOPOZ on August 18th 2023: A New Treatment for CHAPLE Disease
What is VEOPOZ prescribed for? VEOPOZ is prescribed to treat patients 1 year old and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease. What is the name of the drug and what does it do? The drug, VEOPOZ, pronounced VEE-oh-poz, (generic name: pozelimab-bbfg)is an innovative treatment recently approved by the FDA to...