Drugs
Alnylam Pharmaceuticals says it is planning an NDA submission late this year for patisiran, indicated for treating transthyretin-mediated amyloidosis with cardiomyopathy after reporting positive data from the APOLLO-B Phase 3 study. The study met the primary endpoint of change from baseline in the Six-Minute Walk Test at
Drugs
A 9/7-9/9/21 FDA inspection at the DSP Skin Care drug manufacturing facility in Los Angeles, CA, found significant violations of current good manufacturing practice (CGMP) requirements for finished pharmaceuticals and multiple other violations. A 7/19 Warning Letter lists specific violations as: * failing to have for
Drugs
Allarity Therapeutics says its board has ordered a refocus of the company’s oncology drug pipeline away from monotherapies and toward combination drugs. One reason for the shift, it says, is information from a meeting with FDA on a potential Phase 3 clinical development path for dovitinib as a monotherapy
Drugs
FDA has published a draft guidance, Electronic Submission of Expedited Safety Reports from IND-Exempt BA/BE Studies, containing instructions for the electronic submission of expedited individual case safety reports (ICSRs) from IND-exempt bioavailability/bioequivalence studies through the FDA adverse event reporting system database. The document says an ICSR
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An Amgen analysis published in Therapeutic Innovation & Regulatory Science, says the FDA accelerated approval program for drugs “has been largely successful, with half of accelerated approvals converted to traditional approval in a median time of 3.2 years.” The researchers say recent FDA actions show the agency is appropriately
Drugs
FDA has published 29 new and 19 revised product-specific guidances (PSGs) with recommendations for developing generic drugs and generating the evidence needed to support ANDA approval. A Federal Register notice says there are 28 PSGs for products with no approved ANDAs (16 non-complex and 12 complex products). There
Drugs
FDA has placed a clinical hold on Beam Therapeutics’ BEAM-201 and its use in treating relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL)/T cell lymphoblastic lymphoma (T-LL). The company says it filed an IND at the end of June for the specific anti-CD7, multiplex-edited,
Drugs
FDA has approved Janssen’s Stelara (ustekinumab) for treating pediatric patients six years of age and older with active psoriatic arthritis. The human monoclonal antibody is said to work by selectively inhibiting both interleukin (IL)-12 and IL-23, two cytokines thought to play a role in tempering the overactive
Drugs
AstraZeneca has told development partner Innate Pharma that it is discontinuing a monalizumab study after a planned futility interim analysis of the INTERLINK-1 Phase 3 study, sponsored by AstraZeneca, did not meet a pre-defined threshold for efficacy. The INTERLINK-1 study was evaluating monalizumab in combination with cetuximab
Drugs
FDA has published a final guidance entitled “Changes to Disposable Manufacturing Materials: Questions and Answers.” The document describes chemistry, manufacturing, and controls post-approval changes related to disposable manufacturing materials that applicants can use in drug and biological product manufacturing. It addresses questions the agency has received about the limited
Drugs
FDA has granted Omeros an orphan drug designation for OMS906 and its use in treating paroxysmal nocturnal hemoglobinuria (PNH). The rare, life-threatening disease is characterized by red blood cell destruction, blood clots and impaired bone marrow function. The company says that based on OMS906’s mechanism of action as
Drugs
FDA commissioner Robert Califf warned 7/28 that FDA employees affected by user fee funds could start getting layoff notices in early September if House and Senate leaders don’t complete the user fee reauthorization legislative package in the coming weeks. Lawmakers are at a standstill over language added to
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Attorney Daniel Feith (Sidley Austin) says a recent New Jersey federal court decision in a failure-to-warn case involving Merck’s Fosamax (alendronate sodium) helps industry broaden preemption of such claims. Writing in a Washington Legal Foundation Legal Backgrounder, Feith says the case was returned to the New Jersey
Drugs
The National Cancer Institute (NCI) says researchers at the biotechnology company Xilis have used micro-organospheres to accurately predict in a small study of eight colorectal cancer patients whether oxaliplatin would shrink their tumors. The researchers, funded in part by NCI, created the micro-organospheres with tissue from a patient’
Drugs
FDA has accepted for review an Acer Therapeutics NDA resubmission for ACER-001 (sodium phenylbutyrate) for oral suspension for treating patients with urea cycle disorders (UCDs). The resubmission addresses items raised by the agency in a complete response letter issued last month, which identified the company’s third-party contract
Drugs
A bill that would establish a task force between the U.S. Patent and Trademark Office (USPTO) and FDA to improve communication and coordination in implementing each agency’s activities related to drug patents has been advanced by voice vote in the Senate Judiciary Committee. The Interagency Patent Coordination and
Drugs
Lawmakers, patient advocates, and others are continuing to raise questions about a long-delayed review of prescription painkillers promised by FDA commissioner Robert Califf when he was commissioner in 2016, according to an Associated Press report. The story also says Califf commissioned a National Academy of Sciences (NAS) report that
Drugs
FDA is listing the accomplishments from the first year’s work of the Generic Drug Cluster, a multi-country forum working toward a common understanding of each member agency’s generic drug regulatory requirements and toward increased scientific alignment. An online report says the cluster meetings “enabled an agreement for
Drugs
Researchers in the U.S. and Australia say that while there has been a substantial increase in individual participant data (IPD) sharing for industry-sponsored oncology trials in the past five years, much more needs to be done. An investigation published in JAMA Oncology found that of the 304 trials
Drugs
FDA has accepted for review a BLA from ImmunityBio for its N-803 antibody cytokine fusion protein to treat patients with BCG-unresponsive non-muscle-invasive bladder cancer (NMBIC) carcinoma in situ with or without Ta or T1 disease. The company says the submission is based on positive QUILT 3.
Drugs
FDA has accepted for review a Lexicon Pharmaceuticals NDA resubmission for sotagliflozin, an investigational dual SGLT1 and SGLT2 inhibitor for treating heart failure. The agency set a 5/2023 user fee review action date. The company says the submission seeks the following indications: * To reduce the risk of cardiovascular death,
Drugs
Five key Senate leaders have introduced the Cannabis Administration and Opportunity Act (CAOA) to “end the harmful and out-of-touch federal prohibition on cannabis by removing cannabis from the federal list of controlled substances and empowering states to implement their own cannabis laws.” In a statement, the senators say