The FDA has approved Novimmune SA’s Gamifant (emapalumab) for the treatment of pediatric (newborn and above) and adult patients with primary hemophagocytic lymphohistiocytosis (HLH) who have refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. This FDA approval is the first for a drug specifically for HLH. “Primary HLH is a rare and…
FDA Approves First-Line Treatment for Peripheral T-Cell Lymphoma Under New Review Pilot
The U.S. Food and Drug Administration today expanded the approved use of Adcetris (brentuximab vedotin) injection in combination with chemotherapy for adult patients with certain types of peripheral T-cell lymphoma (PTCL). This is the first FDA approval for treatment of newly diagnosed PTCL, and the agency used a new review program to complete the approval…
FDA Approves Jazz Pharmaceuticals’ Xyrem for Treatment of Pediatric Narcolepsy
The FDA approved Xyrem (sodium oxybate) for the treatment of cataplexy and excessive daytime sleepiness (EDS) in pediatric patients (seven to 17 years old) with narcolepsy. Xyrem is a central nervous system (CNS) depressant that was approved in 2002 for the treatment of cataplexy in adult patients with narcolepsy. Cataplexy is a sudden and transient episode…
FDA Approves Antibacterial Drug to Treat Serious Lung Disease Using a Novel Pathway
The FDA approved a new drug, Arikayce (amikacin liposome inhalation suspension), for the treatment of lung disease caused by a group of bacteria, Mycobacterium avium complex (MAC) in a limited population of patients with the disease who do not respond to conventional treatment (refractory disease). MAC is a type of nontuberculous mycobacteria (NTM) commonly found…
FDA Approves AstraZeneca’s Treatment for Hairy Cell Leukemia
The FDA approved Lumoxiti (moxetumomab pasudotox-tdfk) injection for intravenous use for the treatment of adult patients with relapsed or refractory hairy cell leukemia (HCL) who have received at least two prior systemic therapies, including treatment with a purine nucleoside analog. Lumoxiti is a CD22-directed cytotoxin and is the first of this type of treatment for…
FDA Approves New Treatment For Rare Genetic Disorder
Amicus Therapeutics has received approval for Galafold (migalastat), the first oral medication for the treatment of adults with Fabry disease. The drug is indicated for adults with Fabry disease who have a genetic mutation determined to be responsive (“amenable”) to treatment with Galafold based on laboratory data. Fabry disease is a rare and serious genetic…
FDA Approves Onpattro, First-Of-Its Kind Targeted RNA-Based Therapy
The FDA approved Onpattro (patisiran) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients. This is the first FDA-approved treatment for patients with polyneuropathy caused by hATTR, a rare, debilitating and often fatal genetic disease characterized by the buildup of abnormal amyloid protein in peripheral nerves,…
FDA Approves First Generic Under New Pathway To Enhance Market Competition
The FDA approved several strengths of potassium chloride oral solution as the first generic drugs to receive a Competitive Generic Therapy (CGT) designation. This new approval pathway was created to expedite the development and review of a generic drug for products that lack competition. Potassium chloride is an oral treatment that is indicated for the treatment…
FDA Approves Treatment For Two Rare Types Of Non-Hodgkin Lymphoma
The FDA approved Poteligeo (mogamulizumab-kpkc) injection for intravenous use for the treatment of adult patients with relapsed or refractory mycosis fungoides (MF) or Sézary syndrome (SS) after at least one prior systemic therapy. This approval provides a new treatment option for patients with MF and is the first FDA approval of a drug specifically for SS.…
FDA Approves Pfizer’s Biosimilar To Neupogen
FDA approved Pfizer’s Nivestym (filgrastim-aafi), a biosimilar to Neupogen (filgrastim). This is the second approved biosimilar to Neupogen approved in the U.S. Nivestym is approved for the same indications as Neupogen, and can be prescribed by a health care professional for: • patients with cancer receiving myelosuppressive chemotherapy; • patients with…
FDA Approves Agios Pharmaceuticals’ Tibsovo
The U.S. Food and Drug Administration today approved Tibsovo (ivosidenib) tablets for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) who have a specific genetic mutation. This is the first drug in its class (IDH1 inhibitors) and is approved for use with an FDA-approved companion diagnostic used to detect specific…
Venclexta-Rituxan Combo Approved For Chronic Lymphocytic Leukemia Or Small Lymphocytic Lymphoma
The approval of Genentech and AbbVie’s Venclexta was based on Murano (NCT02005471), a randomized (1:1), multicenter, open-label trial of venetoclax with rituximab (VEN+R) versus bendamustine with rituximab (B+R) in 389 patients with CLL who had received at least one prior line of therapy. Patients in the VEN+R arm completed a 5-week ramp-up venetoclax scheduleand then…
FDA Approves First Therapy For Rare Inherited Form Of Rickets
Crysvita (burosumab-twza), the first drug approved to treat adults and children ages 1 year and older with x-linked hypophosphatemia (XLH), a rare, inherited form of rickets, has been approved by the FDA. XLH causes low levels of phosphorus in the blood. It leads to impaired bone growth and development in children and adolescents and problems with…
FDA Approves New Treatment For Thrombocytopenia In Adults With ITP
The FDA approved fostamatinib disodium hexahydrate tablets (Tavalisse, Rigel Pharmaceuticals, Inc.) for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. The most common adverse reactions in at least five percent of patients treated with fostamatinib were diarrhea, hypertension, nausea, dizziness, alanine…
FDA Approves New Indication For Blincyto
The FDA granted accelerated approval to Blincyto (blinatumomab) to treat adults and children with B-cell precursor acute lymphoblastic leukemia (ALL) who are in remission but still have minimal residual disease (MRD). MRD refers to the presence of cancer cells below a level that can be seen under the microscope. In patients who have achieved remission after…
FDA Expands Approval Of Adcetris For Treatment Of Hodgkin Lymphoma
The FDA approved Adcetris (brentuximab vedotin) to treat adult patients with previously untreated stage III or IV classical Hodgkin lymphoma (cHL) in combination with chemotherapy. “Today’s approval represents an improvement in the initial treatment regimens of advanced Hodgkin lymphoma that were introduced into clinical practice more than 40 years ago,” said Richard Pazdur, M.D., director of…
FDA Approves Trogarzo For Treatment Of HIV-1 Infection
The FDA approved Trogarzo (ibalizumab-uiyk), a human immunodeficiency virus type 1 (HIV-1) antiretroviral drug, for the treatment of HIV-1 infection in heavily treatment-experienced adults with multidrug resistant HIV-1 infection failing their current antiretroviral regimen. The approved recommended dosage of Trogarzo is a single intravenous (IV) loading dose of 2,000 mg followed by a maintenance IV dose of…