Biologics
FDA has approved a Sanofi Pasteur BLA for Dengvaxia, the first vaccine approved for preventing dengue disease caused by all dengue virus serotypes (1, 2, 3 and 4) in people ages nine through 16 who have laboratory-confirmed previous dengue infection and who live in endemic areas. Dengue is endemic
FDA Policy/General
FDA has issued a draft guidance, Initiation of Voluntary Recalls Under 21 CFR part 7, Subpart C, on the timely initiation of voluntary recalls of FDA-regulated products. The document discusses what preparations firms in a distribution chain, including manufacturers and distributors, should consider making to establish recall initiation procedures;
Biologics
CBER director Peter Marks says one of the highlights for FY 2018 was approval of two gene therapies and three vaccines. The Center’s annual report from the director says the gene therapies were Luxturna, the first directly-administered gene therapy for a specific genetic disorder (an inherited retinal disease)
Biologics
FDA has agreed that Mesoblast can submit on a rolling basis a BLA for its allogeneic cellular medicine remestemcel-L for use in children with steroid-refractory acute Graft Versus Host Disease (SR-aGVHD). Mesoblast says it will submit each BLA module on a rolling basis as it is completed.
Biologics
FDA has accepted a Novartis BLA for brolucizumab (RTH258) for treating wet age-related macular degeneration (AMD), also known as neovascular AMD. The company used a six-month priority review voucher to expedite FDA’s review. The submission is based on Phase 3 data from the HAWK and HARRIER trials.
Biologics
Celgene has submitted a BLA for luspatercept, an erythroid maturation agent for treating adult patients with very low to intermediate risk myelodysplastic syndromes-associated anemia who have ring sideroblasts and require red blood cell (RBC) transfusions. It is also seeking approval for treating adult patients with beta-thalassemia-associated anemia
Biologics
FDA has released the FDA-483 issued following a 9/17-9/21/18 agency inspection at Liveyon, a Yorba Linda, CA, “own label stem cell distributor.” The inspection observations were: * adverse reactions that involved a communicable disease related to an HCT/P (human cells, tissues, or cellular or tissue-
Biologics
FDA sent a 3/29 Warning Letter to Cord for Life, Altamonte Springs, FL, for manufacturing unapproved umbilical cord blood products in violation of current good manufacturing practice (CGMP) requirements, including failing to validate processes to prevent bacterial contamination, raising potential significant safety concerns that put patients at risk. In
Biologics
Novartis says its Sandoz drug unit has resubmitted its biosimilar pegfilgrastim (Amgen’s Neulasta) BLA to address a 6/2016 complete response letter (see earlier story). The resubmission, the company says, includes new data from a pivotal pharmacokinetics and pharmacodynamics study. “This was a single-dose, three-period cross-over
Biologics
Janssen Pharmaceutical has submitted a supplemental BLA seeking approval of Darzalex (daratumumab) in combination with bortezomib, thalidomide and dexamethasone for newly diagnosed patients with multiple myeloma who are eligible for autologous stem cell transplant. The submission is based on data from the Phase 3 CASSIOPEIA (MMY3006) clinical study, which Janssen
Biologics
FDA commissioner Scott Gottlieb and CBER director Peter Marks say the agency is seeking better safety measures to mitigate potential risks to blood products. In a joint statement, they say that technology exists to potentially transform the assurance of blood product safety while also potentially reducing the cost. Whether an
Biologics
FDA has released a final guidance on “Standards Development and the Use of Standards in Regulatory Submissions Reviewed in the Center for Biologics Evaluation and Research.” The document provides CBER recommendations on the use of standards in product development and control as well as the use of such standards in
Biologics
FDA has approved a Genentech sBLA for Tecentriq (atezolizumab) for combination use with carboplatin and etoposide (chemotherapy) for first-line treatment of adults with extensive-stage small cell lung cancer. Approval was based on data from the Phase III IMpower133 study, which showed that Tecentriq in combination with chemotherapy helped
Biologics
Spectrum Pharmaceuticals has withdrawn a BLA for Rolontis (eflapegrastim), a treatment for chemotherapy-induced neutropenia in patients undergoing myelosuppressive chemotherapy, after FDA requested additional manufacturing-related information. The company says it plans to submit a revised BLA as soon as possible. FDA’s review did not cite concerns related to
Biologics
FDA has just released the FDA-483 issued following a 6/18-6/22/18 inspection at Genetech, a San Diego, CA, umbilical cord processing facility. The 10 inspection observations are: * the eligibility of an HCT/P donor was not determined and documented by a responsible person, based on results
Biologics
FDA has agreed to accept a CytoDyn rolling BLA submission for PRO 140 (leronlimab), a CCR5 antagonist, indicated for blocking HIV-1 from entering and infecting immune cells. Previously, the agency granted a fast track designation for leronlimab as a combination therapy with highly active antiretroviral therapy for HIV-infected
Biologics
FDA officials say the agency lacks the resources needed to pursue a comprehensive crackdown on the growing private stem cell industry. Responding to a lengthy Washington Post investigative report reviewing cases of several people who have been hospitalized after being injected with products made from umbilical cord blood, FDA commissioner
Biologics
CBER has issued a guidance, Providing Lot Release Protocol Submissions to the Center for Biologics Evaluation and Research (CBER) in Electronic Format, to assist manufacturers of biological products regulated by CDER to submit lot release protocols in electronic format. The document updates and supercedes a 2007 guidance. The agency says
Biologics
FDA has approved a Novo Nordisk BLA for Esperoct (turoctocog alfa pegol) for treating adults and children with hemophilia A. The therapy is indicated for routine prophylaxis to reduce the frequency of bleeding episodes, on-demand treatment and control of bleeding episodes and perioperative management of bleeding. Approval is based
Biologics
After 16 years at FDA, CDER Therapeutic Biologics and Biosimilars Staff director Leah Christl is leaving to pursue other opportunities, effective 2/22. She joined FDA in 2003 as a regulatory project manager in the Over-the-Counter Drug Products Division. She later served as associate director for regulatory affairs
Biologics
Attorney Mark Schwartz (Hyman, Phelps & McNamara) says FDA has a number of enforcement options it can turn to after its enforcement discretion for stem cell facilities and certain other HCT/P manufacturers ends 11/2020. Writing in his firm’s FDA Law Blog, Schwartz says FDA indicated in a
Biologics
FDA has approved Merck’s Ontruzant (trastuzumab-dttb) as a biosimilar to Genentech’s Herceptin (transtuzumab) for patients with HER2-overexpressing breast cancer. The product will be manufactured by Samsung Bioepis. The Ontruzant prescribing information says common expected side effects include headache, diarrhea, nausea, chills, fever, infection, congestive heart failure,