Drugs
Members of the FDA Central Nervous System Drugs Advisory Committee have voted unanimously to recommend approval of GW Pharma’s Epidiolex, which contains cannabidiol, for treating seizures associated with two rare types of childhood epilepsy — Lennox-Gestaut syndrome and Dravet syndrome. The company says that if FDA approves the drug,
Drugs
FDA medical reviewers say they have concerns about the safety of Lilly’s Olumiant (baricitinib) being proposed to treat adults with moderately to severely active rheumatoid arthritis who have had an inadequate response or are intolerant to methotrexate. The company’s NDA will be considered at a 4/23 meeting
Drugs
FDA has expanded approval for AstraZeneca’s Tagrisso (osimertinib) for the first-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have epidermal growth factor receptor (EGFR) mutations (exon 19 deletions or exon 21 L858R mutations), as detected by an FDA-approved test. Approval was
Drugs
FDA has published an International Council for Harmonization guidance, Q7 Good Manufacturing Practice Guidance for Active Pharmaceutical Ingredients—Questions and Answers, to clarify uncertainties due to interpretation of certain sections of the ICH Q7 guidance on good manufacturing practice for active pharmaceutical ingredients (API). The document says the Q7 guidance
Drugs
FDA has approved a Rigel Pharmaceuticals NDA for Tavalisse (fostamatinib disodium hexahydrate) for treating thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. The oral spleen tyrosine kinase inhibitor is designed to target the underlying autoimmune cause of the disease
Drugs
FDA has released the FDA-483 issued following a 3/12-4/6 inspection at Coastal Meds, a Nashville, TN, outsourcing facility. Specific observations listed on the form were: * aseptic processing areas are deficient regarding the system for monitoring environmental conditions; * protective apparel is not worn as necessary to protect
Drugs
FDA has granted fast track status to Calithera Biosciences for CB-839 and its use in combination with cabozantinib for treating patients with metastatic renal cell carcinoma who have received one or two prior lines of therapy, including at least one vascular endothelial growth factor tyrosine kinase inhibitor or the
Drugs
Speaking 4/17 at the National Policy and Advocacy Summit on Biologics and Biosimilars, CDER Office of New Drugs associate director Leah Christl said that over time the U.S. will see a dramatic increase in the number of biosimilar approvals, based on the quantity of applications that the agency
Drugs
FDA says a draft guidance, Metered Dose Inhaler (MDI) and Dry Powder Inhaler (DPI) Drug Products—Quality Considerations, is available. A 4/19 Federal Register notice says the guidance’s purpose is to provide recommendations to industry in the development and manufacture of inhalation aerosols. The draft describes points to
Drugs
A 7/24-7/26/17 FDA inspection at McCallum Manufacturing, South Yorkshire, UK, found significant violations of current good manufacturing practice (CGMP) regulations for finished pharmaceuticals, according to a 4/9 Warning Letter. Specific violations cited in the letter were: * failing to have, for each batch of drug product,
Drugs
FDA medical reviewers say three clinical trials conducted by GW Pharma “provide substantial evidence of the effectiveness of CBD (cannabidiol) for the treatment of seizures associated with Lennox-Gestaut syndrome (LGS) and Dravet syndrome (DS) in patients two years of age and older.” The assessment is included in a briefing
Drugs
A 9/25-9/28/17 FDA inspection at Luen Wah Medicines, Hong Kong, found significant violations of current good manufacturing practice regulations (CGMP) for finished pharmaceuticals, according to a 4/6 Warning Letter. Specific violations cited in the letter were: * failing to perform, for each batch of drug product,
Drugs
A bipartisan group of four senators has introduced legislation they say will help bring non-opioid or non-addictive pain medications and treatments for substance abuse disorders to patients more quickly. The bill was introduced by Sens. Orrin Hatch (R-UT), Michael Bennet (D-CO), Joe Donnelly (D-IN) and
Drugs
FDA has approved an Ultragenyx Pharmaceutical NDA for Crysvita (burosumab), the first drug approved to treat adults and children ages one year and older with x-linked hypophosphatemia. The rare, inherited form of rickets causes low levels of phosphorus in the blood, and it leads to impaired bone growth and
Drugs
FDA says it has qualified the use of a patient-reported outcome instrument for exploratory use to measure symptoms of non-small cell lung cancer. Before using the “Qualification of Non-Small Cell Lung Cancer Symptom Assessment Questionnaire,” the agency recommends that sponsors should engage the review division early and
Drugs
FDA has released a draft guidance for industry entitled “Investigational In Vitro Diagnostics in Oncology Trials: Streamlined Submission Process for Study Risk Determination.” The document, which was developed by the agency’s Oncology Center of Excellence, CDER, CBER and CDRH. It describes an optional streamlined submission process for determining whether
Drugs
Rep. Frank Pallone, Jr. (D-NJ) has introduced the National Centers of Excellence in Continuous Manufacturing Act of 2018, which would allow FDA to partner with universities across the country to designate them as National Centers of Excellence in Continuous Pharmaceutical Manufacturing. He announced the bill’s introduction during a
Drugs
FDA has rescinded an Alkermes refuse-to-file letter issued late last month on its NDA for ALKS 5461 (buprenorphine and samidorphan) an investigational drug for adjunctive treatment of major depressive disorder in patients with an inadequate response to standard antidepressant therapies. In doing so, the agency has now accepted
Drugs
A new FDA pilot program aims to evaluate model-informed drug development (MIDD) approaches that could “improve clinical trial efficiency, increase the probability of regulatory success, and optimize drug dosing/therapeutic individualization in the absence of dedicated trials,” according to an agency notice. As part of the 2017 renegotiated drug
Drugs
FDA has released the FDA-483 issued following a 5/22-26/17 and 5/29-6/2/17 inspection at the Celltrion drug manufacturing facility in Incheon, Korea. The 12 inspection observations were: * · failing to thoroughly review any unexplained discrepancy, whether or not the batch has already been distributed;
Drugs
FDA has finalized a guidance on Special Protocol Assessment (SPA) that provides drug manufacturers with information about the procedures and policies adopted by CDER/CBER for reaching an agreement on the design and size of certain clinical trials that could support marketing approval. “An SPA agreement indicates concurrence by the
Drugs
FDA has alerted health care professionals about a recall of all non-expired products marketed as sterile made by Coastal Meds (Biloxi, MS) due to visible particles in some drug vials for injection. “Injection of a drug product containing particulate matter may result in serious and potentially life threatening adverse