Drugs
The Tufts Center for the Study of Drug Development says rare disease drug development is one of the most active and fastest growing areas in drug research and development, with nearly one-third of all products in the global R&D pipeline targeting treatments for rare disease indications. But
Drugs
A 10/31-12/18/18 FDA inspection at U.S. Pharmaceuticals, Belleville, NJ, found significant violations of current good manufacturing practice (CGMP) regulations for finished pharmaceuticals. A just-released 6/6 Warning Letter says specific violations were: * failing to thoroughly investigate any unexplained discrepancy or failure of a batch
Drugs
FDA has issued a guidance, Risk Evaluation and Mitigation Strategies: Modifications and Revisions, with information on how the agency defines the types of changes to approved risk evaluation and mitigation strategies (REMS), and how it will process submissions from application holders for changes to REMS. The document specifically provides information
Drugs
FDA has accepted for review six Merck supplemental BLAs to update the dosing frequency for Keytruda (pembrolizumab) to include an every-six-weeks option. Merck says it is seeking approval of a 400 mg every-six-weeks dose infused over 30 minutes for indications in melanoma, classical Hodgkin lymphoma, primary
Drugs
FDA has released the FDA-483 issued following a 5/27-6/4 inspection at Indoco Remedies, Vasco Da Gama, Goa, India. The four specific inspection observations were: * determinations of conformance to appropriate written specifications for acceptance are deficient for drug products; * investigations of a failure of a batch or
Drugs
A 7/23-8/30/18 FDA inspection at the Akorn drug manufacturing facility in Somerset, NJ, found significant violations of current good manufacturing practice (CGMP) regulations for finished pharmaceuticals that were similar to those found in inspections at other Akorn facilities. A just-released 6/13 Warning Letter says
Drugs
A 2/4-2/9 FDA inspection at the Aurobindo Pharma drug manufacturing facility in Ranasathalam (Mandal), India, found significant deviations from current good manufacturing practice (CGMP) regulations for active pharmaceutical ingredients (APIs). A 6/20 Warning Letter says specific deviations were: * failing to extend investigations to other batches that
Drugs
FDA says it has issued Warning Letters to three repackers of active pharmaceutical ingredients (APIs) for significant violations of current good manufacturing practice (CGMP) requirements. An agency news release says the letters went to B&B Pharmaceuticals, Inc. and Asclemed USA, Inc., doing business as Enovachem and Spectrum Laboratory
Drugs
FDA has issued an “immediate effect” guidance, Compliance Policy for Certain Compounding of Oral Oxitriptan (5-HTP) Drug Products for Patients with Tetrahydrobiopterin (BH4) Deficiency, giving the conditions under which the agency does not generally intend to take regulatory action against a licensed pharmacist in a state-licensed pharmacy or
Drugs
FDA has published a draft guidance, Drug Abuse and Dependence Section of Labeling for Human Prescription Drug and Biological Products—Content and Format, to assist applicants in writing the Drug Abuse and Dependence section of labeling, as described in the regulations for the content and format of labeling for human
Drugs
FDA has published a draft guidance, Instructions for Use—Patient Labeling for Human Prescription Drug and Biological Products and Drug-Device and Biologic-Device Combination Products—Content and Format, with recommendations for developing the content and format of an Instructions for Use (IFU) document for human prescription drug and biological
Drugs
Fresenius Kabi USA has recalled two lots of fluorouracil injection, USP 5g/100ml (50mg/ml), 100ml fill in 100ml vials, due to the potential for them to contain glass particulates. “Products containing glass particulate should not be administered intravenously due to the potential for life-threatening consequences,” a recall notice
Drugs
Under a 2017 CDER Manual of Policies and Procedures (MAPP), Prioritization of the Review of Original Abbreviated New Drug Applications (ANDAs), Amendments, and Supplements, ANDA submissions for drug products indicated for the emergency treatment of known or suspected opioid overdose are eligible for priority review. FDA says that based on
Drugs
A 7/6 New York Times editorial board opinion piece urges the Trump Administration to curb drug price increases the way Europe does – “by tying a drug’s price to its actual value.” It says HHS should evaluate and rank medications based on their cost effectiveness. The board notes that
Drugs
On the eve a rule was to go into effect requiring drug pricing information in direct-to-consumer ads, the Washington, DC federal district court ruled 7/8 that HHS lacks the statutory authority under the Social Security Act to mandate such disclosures. The 6/14 complaint from Merck, Eli
Drugs
FDA has posted a final guidance entitled “Epidermolysis Bullosa (EB): Developing Drugs for Treatment of Cutaneous Manifestations.” The document focuses on drug development and trial design issues specific to the treatment of EB, including FDA’s current thinking on trial endpoints. The agency notes that there is not yet sufficient
Drugs
FDA has approved Janssen Pharmaceutical Companies’ Darzalex (daratumumab) in combination with lenalidomide and dexamethasone for treating patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant. Approval was based on data from the Phase 3 MAIA (MMY3008) clinical study, which showed that the combination significantly reduced
Drugs
FDA is proposing a new rule that would enable the seamless transition of some biologics approved under the Federal Food, Drug, and Cosmetic Act (FFDCA) to licensed biologics under the Public Health Service Act. The proposed rule would amend agency regulations on the use of drug master files to allow
Drugs
FDA has published an International Council on Harmonization guidance, M10 Bioanalytical Method Validation, with recommendations for the validation of bioanalytical assays for chemical and biological drug quantification and their application in the analysis of study samples. “Adherence to the principles presented in this guideline will improve the quality and consistency
Drugs
FDA has posted two videos discussing the Complex Innovative Trial Design pilot meeting program to support the goal of facilitating and advancing the use of complex adaptive, Bayesian, and other novel clinical trial designs. The videos introduce the program and discuss its process. The post says the pilot is designed
Drugs
FDA has published a draft guidance, Treatment for Heart Failure: Endpoints for Drug Development, giving the agency’s current thinking about developing drugs to treat heart failure and highlighting remaining areas of uncertainty to encourage discussion and alternative approaches. The document’s purposes are to (1) make it clear that
Drugs
Under a contract with CDER, EdjAnalytics, a data science firm providing artificial intelligence, machine learning and advanced analytics for life sciences, has developed a predictive model to support the agency’s decision-making process to select manufacturing sites for inspection. “The model provides the likelihood of a facility having an