Regulatory Filings and Submissions

Sandoz’s marketing authorization applications for a proposed biosimilar to denosumab are supported by a comprehensive analytical and clinical data package.

FDA has approved the first topical gene therapy product for treating wounds in patients with dystrophic epidermolysis bullosa.

FDA has approved AbbVie’s EPKINLY (epcoritamab-bysp), a bispecific antibody for treating relapsed or refractory diffuse large B-cell lymphoma.

The UK’s National Institute for Health and Care Excellence has recommended dapagliflozin (Forxiga) from AstraZeneca as a treatment option for adults with symptomatic chronic heart failure with preserved or mildly reduced ejection fraction.

UK’s NICE has recommended Kapruvia as a treatment for adult patients with moderate-to-severe chronic kidney disease (CKD)-associated pruritus.

UK's NICE has recommended RINVOQ (upadacitinib) as a treatment option for adult patients in England and Wales with moderately to severely active Crohn’s disease.

The two draft guidance documents provide recommendations for the approval of pediatric drugs, biologics, and vaccines under the Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act.

EC has granted marketing authorization to Chiesi Global Rare Diseases and Protalix BioTherapeutics for PRX-102 (pegunigalsidase alfa).

The recent revision is intended to provide guidance on QRM principles and tools that can be used for different aspects of pharmaceutical quality.

FDA has granted fast track designation to Caribou Biosciences for its allogeneic CAR-T cell therapy for relapsed or refractory multiple myeloma.

Will court challenges to FDA’s oversight authority open the door for multiple lawsuits to block agency decisions on all sides?

FDA aims to address mounting criticisms of its accelerated approval pathway.

Takeda received approval in Japan for a subcutaneous injection formulation of Entyvio (vedolizumab) for treating ulcerative colitis.

FDA has approved Acadia Pharmaceuticlas’ Daybue (trofinetide), the first treatment for Rett syndrome, a rare genetic disorder.

FDA has approved Pfizer’s ZAVZPRET (zavegepant), a new nasal spray treatment for migraine.

Even after an FDA advisory panel voted 14–1 in October 2022 to remove the drug, Makena manufacturer Covis Pharma continued to press for some continued access to the therapy.

Stepwise paediatric investigation plans aim to boost the development of medicines for children.

This warning is the second on top of a previous warning not to purchase or use EzriCare Artificial Tears or Delsam Pharma’s Artificial Tears due to potential bacterial contamination.

Apellis Pharmaceuticals’ Syfovre is a pegcetacoplan injection intended for the treatment of geographic atrophy secondary to age-related macular degeneration.

The intention of the FOA is to address diverse topics related to improving the efficiency of biosimilar product development and advancing the development of interchangeable products.

The COVID-19 pandemic helped to showcase RNA molecules and their therapeutic potential. There were also lessons learned in terms of drug delivery, manufacturing, logistics, and storage.

Clinicians, pediatric patients, regulators, and more are being considered to further strengthen focus on unmet medical needs.

A Congressional probe of FDA’s approval of Alzheimer’s treatment targets the agency’s interactions with drug manufacturers.

In a response letter, FDA stated that it could not grant Eli Lilly and Company accelerated approval due to concerns surrounding insufficient quantity of clinical trial data.

The guidance provides examples of required and recommended information in the Dosage and Administration section.

Does the new regulatory framework have the potential to incentivise R&D investment in rare diseases?

Bylvay has minimal systemic exposure and acts locally in the small intestine as a potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi).

Overall industry growth conceals growing numbers of smaller, faster-paced, adaptable corporate structures.

FDA keeps its user fees but fails to gain important reforms.

New medications show exciting outcomes for weight loss.