Drugs
Acer Therapeutics says it is planning a pivotal clinical trial of its Edsivo (celiprolol) in patients with COL3A1+ vascular Ehlers-Danlos syndrome (vEDS). The company says the agency agreed with its submission of a proposed protocol under a special protocol assessment with a possible breakthrough designation. Minutes from a recent
Drugs
FDA has published a draft guidance, Core Patient-Reported Outcomes in Cancer Clinical Trials, with recommendations to sponsors for the collection of a core set of patient-related clinical outcomes in cancer clinical trials and related considerations for instrument selection and trial design. The document says some of the recommendations
Drugs
FDA has approved a Vertex Pharmaceuticals supplemental NDA for Trikafta (elexacaftor/tezacaftor/ivacaftor) to include children with cystic fibrosis aged 6 through 11 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive
Drugs
FDA and HHS have released a report with policy recommendations to address the vulnerabilities in the nation’s pharmaceutical supply chains. An HHS announcement says the report and its recommendations have been accepted by President Joe Biden. “Last year the American people experienced a widespread and significant shortage of N95
Drugs
Two people who worked for Tellus Clinical Research in Florida pleaded guilty 6/8 in a Florida federal court to participating in a conspiracy to falsify clinical trial data. A Department of Justice statement says that Eduardo Novarro, Miami, FL, a sub-investigator, and Nayade Varona, Port St. Lucie, FL,
Drugs
FDA says its review of a Web site and social media pages maintained by Everything Health LLC, Carnation, WA, determined that the company illegally offers CoronaBox for sale in the U.S. A 5/24 Warning Letter says the product is intended to mitigate, prevent, treat, diagnose, or cure Covid-
Drugs
RVL Pharmaceuticals is petitioning FDA to require ANDA applicants and section 505(b)(2) applicants who seek “A” ratings to use appropriate methods to establish that their proposed products are bioequivalent and therapeutically equivalent to Upneeq (oxymetazoline HCl ophthalmic solution) 0/1%. The company’s 6/2 petition asks the
Drugs
FDA has informed Incyte that it has extended the review period for the company’s sNDA for Jakafi (ruxolitinib) for treating adult and pediatric patients age 12 and older with steroid-refractory chronic graft-versus-host disease. The new PDUFA target action date is 9/22, the agency says. FDA
Drugs
OptiNose is petitioning FDA to take specific actions regarding any ANDA or section 505(b)(2) application that references or relies upon the company’s Xhance (fluticasone propionate) nasal spray. A 5/31 petition asks that the agency: * not approve an ANDA referencing Xhance unless the applicant demonstrates bioequivalence and
Drugs
[Report by Jim Dickinson and David McFarland] FDA’s 6/7 accelerated approval for Biogen’s Alzheimer’s drug Aduhelm (aducanumab-avwa) has provoked an immediate uproar among stakeholders over whether it undermines the agency’s coveted gold standard of scientific objectivity. Striking a positive lead from the legal constituency,
Drugs
FDA says it has decided not to accept a Verily Life Sciences 1/25 Letter of Intent for the Virtual Motor Exam for Parkinson’s disease, Part 3 Estimator, as measured by the Verily Study Watch. An agency determination letter says the letter proposed assessment of motor symptom severity in
Drugs
FDA has granted BerGenBio ASA a fast track designation for bemcentinib in combination with an anti-PD-(L)1 agent for treating patients with AXL-positive advanced/metastatic non-small cell lung cancer. The designation has been granted for patients without actionable mutations, with disease progression on or after treatment
Drugs
FDA has granted accelerated approval for Biogen’s Aduhelm (aducanumab), the first drug since 2003 approved for treating Alzheimer’s. Approval was based on the drug’s effect on a surrogate endpoint — reducing amyloid beta plaque— and a post-approval trial is required to verify that the drug provides the
Drugs
FDA has approved a Medtronic PMA for the SenSight Directional Lead System used for deep brain stimulation (DBS) therapy. The company says the device is designed for treating some symptoms associated with movement disorders like Parkinson's disease, dystonia and essential tremor, and medically refractory epilepsy. DBS comprises a
Drugs
FDA has approved an Alexion Pharmaceuticals BLA for Ultomiris (ravulizumab-cwvz) injection and its use in treating patients aged one month and older with paroxysmal nocturnal hemoglobinuria (PNH). The a rare, life-threatening blood disease is characterized by red blood cell destruction, anemia, blood clots, and impaired bone marrow function.
Drugs
FDA has approved a Novo Nordisk NDA for Wegovy (semaglutide) injection (2.4 mg once weekly) for chronic weight management in overweight adults with at least one weight-related condition (such as high blood pressure, Type 2 diabetes, or high cholesterol). The approval recommends the drug’s use in addition
Drugs
FDA has approved a Chimerix NDA for Tembexa (brincidofovir) for treating smallpox. “Although naturally occurring smallpox no longer exists, concerns about potential uses of variola virus as a bioweapon has made smallpox drug development an important component of the U.S. medical countermeasures response,” an FDA release says. Because smallpox
Drugs
FDA has finalized two guidances and published two draft guidances to help ensure that prescription drugs are identified and traced properly as they move through the supply chain. An agency online notice says the documents provide agency recommendations for how to comply with applicable Drug Supply Chain Security Act (DSCSA)
Drugs
FDA has accepted a PharmaEssentia USA resubmission of a BLA seeking approval for ropeginterferon alfa-2b-njft to treat polycythemia vera (PV), a rare blood cancer. The agency assigned a six-month review timeframe with a PDUFA action date of 11/13. FDA has granted ropeginterferon alfa-2b-njft orphan
Drugs
FDA has granted priority review for an Amryt NDA for oleogel-S10 to treat epidermolysis bullosa. The agency previously granted the treatment fast track designation and rare pediatric disease designation. Amryt says oleogel-S10 is a potential treatment for the cutaneous manifestations of junctional and dystrophic epidermolysis bullosa, a rare
Drugs
Dermavant Sciences has submitted an NDA for tapinarof for treating mild, moderate and severe plaque psoriasis in adult patients. The submission for the therapeutic aryl hydrocarbon receptor modulating agent is based on data from two Phase 3 studies (PSOARING 1 and PSOARING 2) and interim results from PSOARING 3, a
Drugs
FDA has posted a draft guidance entitled Oral Drug Products Administered Via Enteral Feeding Tube: In Vitro Testing and Labeling Recommendations. The document covers in vitro testing recommendations to ensure oral drug product quality and bioequivalence to a reference-listed drug when evaluating a drug product’s suitability for administration