Regulations

FDA rejects early approval for Lilly’s Alzheimer’s drug

The US FDA has declined to grant Eli Lilly accelerated approval of monoclonal antibody drug, donanemab. Eli Lilly said the complete response letter (CRL) received from the US Food and Drug Administration (FDA) has been issued due to the limited number of patients with at least one year of drug exposure data provided in the application and stated that no other deficiencies have been identified. The FDA specifically requested that Lilly provide data from a minimum of 100 patients who…

Eisai and Biogen: FDA approve Alzheimer’s mAb lecanemab

Leqembi (lecanemab) has received accelerated approval for Alzheimer’s disease and will be priced at $26,500 per year, roughly half the original price of Biogen’s controversial drug Aduhelm (aducanumab). The US Food and Drug Administration (FDA) approved Leqembi on Friday for patients with the mild cognitive impairment or mild dementia stage of disease and confirmed presence of Aβ pathology under an accelerated pathway based on reduction in amyloid beta plaques observed in Phase II trial patients. Marketing authorization holder Eisai has…

Ferring gets FDA greenlight for bladder cancer gene therapy

The US Food and Drug Administration (FDA) has approved Ferring Pharmaceuticals’ adenovirus vector-based gene therapy, Adstiladrin (nadofaragene firadenovec-vncg). Adstiladrin is an intravesical therapy administered to the patient at three-month intervals. The therapy is used to treat adults with high-risk Bascillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) that works by targeting the individuals’ bladder wall cells to boost the body’s natural defenses to fight cancer. According to the firm, the FDA approval is based on positive Phase III clinical trial…

Cell and gene therapy regulations moving in right direction says ARM

PDUFA VII will help clarify CMC requirements for cell and gene therapy firms according to the Alliance for Regenerative Medicine (ARM), which says regulations around such products are moving in the right direction. The Prescription Drug User Fee Act (PDUFA) VII – legislation that enables the US FDA to collect use fees from drug developers and strengthens the agency’s review capabilities for small and large molecule drugs and advanced therapeutics – covers the fiscal years 2023 to 2027. Cell and gene…

Lack of AdComs could indicate FDA comfortable with AAV5 safety

CSL’s recent approval and the retraction of an AdCom meeting for Biomarin may demonstrate the US FDA has become comfortable with hemophilia gene therapies and AAV5 vectors. Last week, while much of the US was preparing turkey dinners and bracing itself for family feuds, the US Food and Drug Administration (FDA) was ‘giving thanks’ to gene therapies, specifically against hemophilia. On Tuesday, CSL Behring won approval for Hemgenix (etranacogene dezaparvovec) – a single-dose therapy to treat adults with hemophilia B.…

Biosimilars: Senate Bill aims to change interchangeability requirement

A Republican Senator has introduced the Biosimilar Red Tape Elimination Act in an effort to remove the need for switching studies and increase biologics competition. The arrival of biosimilars in the US came almost a decade after Europe first embraced the competitive biologic modality. And even then, biosimilars have been distinguished in the US due to the need to prove interchangeability with the reference product through switching studies if they are to be substituted without the intervention of a health…

CSL Behring up as FDA approves hemophilia B gene therapy

With a list price of $3.5 million, Hemgenix (etranacogene dezaparvovec) becomes the most expensive single-dose drug and the first gene therapy approved for hemophilia B. The US Food and Drug Administration (FDA) gave CSL Behring’s one-time gene therapy the thumbs up yesterday to treat adults with hemophilia B, a rare bleeding disorder caused by a gene defect that results in insufficient production of blood clotting protein factor IX. “We are thrilled to witness this milestone in hemophilia B treatment,” said…

FDA discusses the ‘must-have’ CMC info to advance gene therapies

Having qualified assays and appropriate cGMPs in place is crucial to support product quality when bringing a gene therapy product into late-phase studies, the US FDA says. This week, the US Food and Drug Administration (FDA) hosted the first in a series of virtual town halls aimed at engaging with product development stakeholders and researchers to discuss topics related to advanced therapies. The event run by the Office of Tissues and Advanced Therapies (OTAT) – soon to become the Office…

Emergent: Compliance underway at Camden, but ‘it’s a long road ahead’

Emergent has invested in quality enhancement and improvement initiatives in response to an FDA warning letter but says there is still much to do. For Emergent BioSolution’s third quarter results, revenues from its contract development and manufacturing organization (CDMO) business unit dropped $76.4 million year-on-year. While much of this was due to lowered sales from COVID-19 vaccine clients AstraZeneca and J&J, the decrease also reflected reduced production at the firm’s Camden facility in Baltimore, Maryland, which recently received a warning…

Bristol Myers hit with warning letter at AZ fill/finish plant

The US FDA highlighted contamination issues at a facility that produces cancer drug Abraxane but has been earmarked by Bristol Myers Squibb to become a biologics drug product plant. The US Food and Drug Administration (FDA) inspected the facility in Phoenix, Arizona from March 28 to April 6, 2022 and issued a warning letter on October 31. The violations noted in the letter included failures to sufficiently investigate contamination issues and to effectively implement corrective actions and preventive actions (CAPA).…