Drugs
FDA Division of Oncology director Harpreet Singh says efforts to streamline oncology clinical trials can produce discomfort in investigators over issues such as safety reporting being not as rigorous as investigators expect FDA to want. Singh was a panelist on an American Society of Clinical Oncology podcast, discussing FDA’s
Drugs
FDA has granted Biogen accelerated approval for Qalsody (tofersen) to treat patients with amyotrophic lateral sclerosis (ALS) associated with superoxide dismutase 1 (SOD1) gene mutation, which in turn is linked with 20% of familial cases and about 2% of sporadic ALS cases. The antisense oligonucleotide, which is designed to target
Drugs
FDA has lifted a clinical hold against a MaaT Pharma IND for an open-label, single arm Phase 3 pivotal clinical trial evaluating the safety and efficacy of MaaT013 to treat gastrointestinal acute Graft-versus-Host Disease as a third line treatment. “This is the first time the agency has
Drugs
Senate Health, Education, Labor, and Pensions (HELP) Committee chairman Bernie Sanders (I-VT) and ranking member Bill Cassidy (R-LA) announced 4/25 that a legislative bipartisan deal has been reached to expand the availability of low-cost generic drugs, clarify orphan drug exclusivity, and reform the pharmacy benefit managers
Drugs
FDA has sent a letter to Sun Pharma telling the company that it is out of compliance with a 2012 consent decree after an inspection last year at the company’s Mohali (Punjab), India cited significant GMP violations and the agency classified the inspection as “Official Action Indicated." “U.
Drugs
The U.S. Supreme Court late 4/21 granted FDA a stay of Texas federal judge Matthew Kacsmaryk’s nationwide ban on mifepristone while it is appealed in the U.S. Court of Appeals for the Fifth Circuit. Oral arguments are on fast track in that venue and are scheduled
Drugs
CDER’s Office of Prescription Drug Promotion (OPDP) is planning a survey to better understand quantitative claims in direct-to-consumer prescription drug advertising. The office notes that DTC advertising does include quantitative claims about a drug’s efficacy or risks and there is existing guidance, “Presenting Quantitative Efficacy and
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Applied Therapeutics plans to request a pre-NDA meeting with FDA on its govorestat (AT-007), a central nervous system penetrant aldose reductase inhibitor for treating patients with galactosemia, after reporting data from a recent Phase 3 study. Govorestat demonstrated “consistent long-term clinical outcomes benefit across a range of
Drugs
FDA has granted Lantheus Holdings and Point Biopharma Global a fast track designation for 177Lu-PNT2002 for treating metastatic castration resistant prostate cancer (mCRPC). The companies say the radiopharmaceutical therapy combines a prostate specific membrane antigen (PSMA)-targeted ligand, PSMA-I&T, with a beta-emitting radioisotope. The therapy
Drugs
After a delay caused by an FDA 2021 inspection and Warning Letter, the agency has approved Medtronic’s MiniMed 780G System that includes modifications to the device’s SmartGuard (SG) Technology and expanding the indications for use to include the Guardian 4 Sensor. The device is intended for “continuous delivery
Drugs
Generic drug lobbying arm Association for Accessible Medicines (AAM) says FDA’s draft guidance entitled “Product-Specific Guidance (PSG) Meetings Between FDA and ANDA Applicants Under GDUFA” does not implement user fee-negotiated commitments regarding teleconferences and meetings as intended, which may deter their use. In just-posted comments to
Drugs
FDA has placed a partial clinical hold on a Foghorn Therapeutics clinical study of FHD-609 in synovial sarcoma and SMARCB1-deleted tumors after the company paused enrollment due to a Grade 4 QTc prolongation event in a synovial sarcoma patient who received the second highest dose. The partial hold
Drugs
FDA has extended by three months its review of a Daiichi Sankyo NDA resubmisson for quizartinib in combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy for treating adult patients with newly diagnosed (FTL3-ITP positive) acute myeloid leukemia (AML). The revised user fee action target date
Drugs
CDER has denied a 2021 citizen petition from Bausch Health that urged FDA not to approve any ANDA that cites Duobrii as the reference-listed drug unless the application meets all the specifications sought by the company. DuobriI (halobetasol propionate and tazarotene) lotion is a topical drug to treat adults
Drugs
FDA has approved Novartis’ Radioligand Therapy (RLT) manufacturing facility in Millburn, NJ to manufacture prostate cancer therapy Pluvicto (lutetium Lu 177 vipivotide tetraxetan), which has been on the agency’s drug shortage list. “Production will begin in the coming weeks and ramp up gradually,” Novartis says. “The site is expected
Drugs
New research on drug direct-to-consumer (DTC) television advertising has (XXX INSERT XXX)found that viewers misinterpreted oncology clinical trial endpoint-related claims that referred to overall survival (OS), overall response rate (ORR), and progression-free survival (PFS). “Although participants differentiated between OS, ORR, and PFS, we found evidence
Drugs
FDA has released the form FDA-483 issued following a 3/21-3/29 inspection at Lupin Limited, a drug manufacturer in Pithampur, India. The 10 inspection observations on the form are: * investigations of a failure of a batch or any of its components to meet any of its specifications
Drugs
Sarepta Therapeutics has submitted comments on an FDA draft product-specific guidance (PSG) for golodirsen intravenous solution, a treatment approved under the accelerated approval pathway for treating Duchenne muscular dystrophy in patients who have a confirmed genetic mutation in the dystrophin gene that is amenable to exon 53 skipping. The
Drugs
An FDA online post describes the work done by agency researchers in the Division of Applied Regulatory Science to develop a new quantitative structure-activity relationship (QSAR) model to predict blood-brain barrier permeability. The post says two statistical-based QSAR models were developed to predict drug permeability across the
Drugs
“Had FDA been enforcing the regulations … up to this point, the U.S. government could have imposed at least $45 billion in fines,” say three Johns Hopkins University students who are members of the Clinical Trials Transparency Committee of the Hopkins chapter of Universities Allied for Essential Medicines (UAEM). “However,
Drugs
DBV Technologies has received written responses from FDA after a pre-BLA meeting in February about the regulatory path for investigational Viaskin Peanut 250 μg patch (DBV712) for use in toddlers with a confirmed peanut allergy. The French biotech company says the agency granted its meeting request on a written
Drugs
BelGene says the results of the Phase 3 RATIONALE 305 trial of tislelizumab with chemotherapy met its primary endpoint of overall survival compared with chemotherapy alone in patients with advanced unresectable or metastatic gastric or gastroesophageal junction adenocarcinoma, regardless of PD-L1 status. A company statement says no new safety