Drugs
A 4/8 BioCryst voluntary decision to pause enrollment in clinical trials of its oral Factor D inhibitor, BCX9930, has transitioned to an FDA partial clinical hold. In a 5/5 quarterly financial report, the company says the hold was related to observed elevations in serum creatinine seen in some
Drugs
Psychiatrist Brian Barnett says the effective and relatively inexpensive schizophrenia drug clozapine “is rarely prescribed in the U.S. largely because of its onerous REMS (Risk Evaluation and Mitigation Strategy) requirements.” Writing in a Stat online opinion piece, Barnett says the REMS is intended to address agranulocytosis, a rare but
Drugs
Harvard University researchers say the percentage of drugs referred for advisory committee review has dropped from 59% in 2011 to 6% in 2021. Writing in Health Affairs, the researchers say meaningful reforms of the advisory committee process could better ensure public trust in an agency decision to allow a drug
Drugs
Pfizer’s Covid-19 treatment Paxlovid (nirmatrelvir tablets and ritonavir tablets) is now widely available in community pharmacies and is authorized for treating mild-to-moderate Covid-19 in adults and pediatric patients age 12 and older with positive results of direct SARS-CoV-2 viral testing who are also
Drugs
The International Council on Harmonization says its Model-Informed Drug Development (MIDD) Discussion Group, which was established in 1/2021, has posted a roadmap with respect to future MIDD-related guidelines. Access the roadmap here.
Drugs
Sens. Maggie Hassan (D-NH) and Mike Braun (R-IN) are calling on FDA commissioner Robert Califf to “update FDA’s opioid labeling practices to reflect current public health and safety knowledge.” In a 4/28 letter, the two cite 2021 opioid death statistics from their states and assert that
Drugs
FDA has lifted a clinical hold on Pfizer’s giroctocogene fitelparvovec (Hemophilia A gene therapy), the company disclosed in a 5/3 earnings announcement. The hold was placed on the Phase 3 AFFINE study 11/3/2021 following observance of Factor VIII levels greater than 150% in some study participants.
Drugs
Writing in a New England Journal of Medicine Perspective piece, CDER executives John Concato and Jacqueline Corrigan-Curay say that more than five years after the passage of the 21st Century Cures Act, the terms real-world data (RWD) and real-world evidence (RWE) continue to be used inconsistently and
Drugs
An 11/1-11/5/21 FDA inspection at a contract testing laboratory operated by the Miami University Department of Chemistry and Biochemistry, Oxford, OH, found significant deviations from current good manufacturing practice (CGMP) for active pharmaceutical ingredients (APIs). A 4/20 Warning Letter says specific deviations were: * failing to
Drugs
FDA has issued HutchMed a complete response letter on its NDA for surufatinib, indicated for treating pancreatic (pNETs) and extra-pancreatic (epNETs) neuroendocrine tumors. The agency letter said that a multi-regional clinical trial (MRCT) is required for U.S. approval, according to the company. “FDA determined that the current
Drugs
FDA has denied an 11/2021 Public Citizen petition calling for the disqualification of two researchers at Hennepin County Medical Center, Minneapolis, MN, who “repeatedly and deliberately violated agency regulations intended to protect human subjects.” The advocacy group said the doctors conducted a series of clinical trials that tested the
Drugs
FDA has issued a complete response letter to Shanghai Junshi Biosciences and Coherus BioSciences on their BLA for toripalimab in combination with gemcitabine and cisplatin in the first-line treatment of patients with advanced recurrent or metastatic nasopharyngeal carcinoma (NPC) and for toripalimab monotherapy in the second-line or later
Drugs
FDA has released a final guidance entitled Electronic Submission of IND Safety Reports Technical Conformance Guide. The document provides specifications, recommendations, and general considerations on how to submit electronic investigational new drug application (IND) safety reports to CDER/CBER. Updates to the guide since its 10/2019 release in draft
Drugs
FDA has placed a clinical hold on a Vertex Pharmaceuticals Phase 1/2 clinical trial of VX-880, an investigational stem cell-derived, fully differentiated pancreatic islet cell replacement therapy for people with Type 1 diabetes with impaired hypoglycemic awareness and severe hypoglycemia. The agency made a determination that there
Drugs
FDA has sent an email to Praxis Precision Medicines placing a clinical hold on PRAX-222, an antisense oligonucleotide for treating patients with SCN2A gain-of-function epilepsy. The company had been planning to begin a seamless study of PRAX-222 this quarter for treating SCN2A developmental and epileptic encephalopathy.
Drugs
CDER’s Office of Compliance (OC) says its priorities for 2022 include supporting FDA’s Covid-19 response (supporting new therapy development and protecting the public from harmful and illegal products), reducing harm caused by opioid crisis, and improving the agency’s oversight of drug compounding operations. In a just-
Drugs
FDA has released a draft guidance entitled Crohn’s Disease: Developing Drugs for Treatment. The document addresses the agency’s current recommendations on clinical trials for Crohn’s Disease (CD) drugs being developed, such as trial population, trial design, efficacy considerations, and safety assessments. FDA notes that the guidance does
Drugs
FDA has lifted a clinical hold on a Finch Therapeutics Group IND for CP101, an orally administered microbiome therapeutic which is in late-stage clinical development for preventing recurrent C. difficile infection (CDI). “The FDA lifted the clinical hold following a review of information Finch provided related to its SARS-
Drugs
FDA has released a draft guidance entitled Ulcerative Colitis: Developing Drugs for Treatment. The document addresses FDA’s current thinking on the necessary attributes of clinical trials for such drugs, including trial population, trial design, efficacy considerations, and safety assessments. The guidance does not address extraintestinal manifestations of ulcerative colitis
Drugs
Simulations Plus, which says it provides modeling and simulation services for the pharmaceutical and biotechnology industries, says it has completed a project funded by FDA and the Center for Research on Complex Generics to establish the suitability of model-integrated evidence to demonstrate bioequivalence in long-acting injectable and implantable
Drugs
FDA is considering asking Congress for additional authority to have the option of requiring drug sponsors in their NDAs to demonstrate their product’s superiority over drugs that are already on the market. During a 4/28 Senate appropriations subcommittee hearing on FDA’s fiscal year 2023 budget request, commissioner
Drugs
The Biotechnology Innovation Organization (BIO) says it agrees with FDA that the rarity of pediatric cancers may render investigations of multiple same-in-class products unfeasible. The comment is included in the trade association’s response to the agency’s pediatric oncology subcommittee of the Oncologic Drugs Advisory Committee’s