Regulatory Authority Actions

FDA’s latest approval of Imfinzi (durvalumab) in combination with chemotherapy for treating resectable non-small cell lung cancer before and after surgery is based on Phase III trial results showing that the regimen reduced the risk of recurrence, progression, or death by 32%.

PiaSky (crovalimab) has previously been approved in countries such as the United States and Japan for monthly, subcutaneous use that may be self-administered with adequate training.

Opaganib was given orphan drug designation for the treatment of neuroblastoma in children, which is rare, but the most common infancy malignancy.

In a 24-week study of patients with PAH, six-minute walking distance improved by an average of 40.8 meters, and risk of death or clinical worsening was reduced by 82%.

The combination of Opdivo plus Yervoy is already established as a second-line treatment for patients with advanced hepatocellular carcinoma.

The International Coalition of Medicines Regulatory Authorities’ new Collaborative Hybrid Inspection Pilot program allows a CDMO to gain approval from multiple countries simultaneously, as opposed to separately phased inspections from different corners of the globe.

Fasenra, which has been approved since 2019 in the United States in a pre-filled, single-use auto-injector form that can be administered either in a doctor’s office or at home, is now approved in China for age 12 and up.

Approximately 15% of all lung cancers diagnosed in the US are of the small-cell variety, and of those, 70% are considered extensive-stage.

Breyanzi demonstrated an overall high response rate in adults with relapsed or refractory follicular lymphoma in a Phase II study.

NexoBrid, a topically administered biological product, is now authorized for use in all age groups in the United States, European Union, and Japan.

Imfinzi may be used alone, or with chemotherapy in tandem with the other approved treatment, Lynparza.

Yorvipath is a prodrug of parathyroid hormone (PTH) (1-34), designed to be administered once daily to provide continuous exposure to released PTH.

AstraZeneca and Daiichi Sankyo first entered into an agreement to develop Enhertu in March 2019, and the antibody-drug conjugate has since been approved for various groups in more than 65 countries.

The reopening of comments addresses the addition of a warning to consumers that acetaminophen may cause skin irritation characterized by reddening, blisters, and rashes.

Synovial sarcoma is a rare form of cancer impacting about 1000 adults in the United States each year, most of them men in their 30s or younger.

The implementation of new quality control methods must be reported to regulators, says Susan J. Schniepp, distinguished fellow at Regulatory Compliance Associates.

An additional 11 medicines were recommended for extension of therapeutic indications, while one negative opinion was issued.

Combination products may be reviewed in a single application or in separate applications for each constituent part, and in rare cases, FDA may decide which type of application process is appropriate.

Other indications for which the Sanofi treatment has been approved include atopic dermatitis, asthma, and chronic rhinosinusitis.

Kisunla (donanemab-azbt) reduced amyloid plaques in Phase III study participants by 84% after 18 months.

EMA’s CHMP gave positive opinions on a nasal delivery for epinephrine and for a first-in-class medicine to treat pulmonary arterial hypertension, among others, including a biosimilar for treatment of autoimmune diseases.

The seven chosen sponsors will help accelerate development of novel drugs and biologics for rare diseases.

Results of a Phase III trial showed that Tagrisso with the addition of chemotherapy reduced the risk of disease progression or death associated with EGFR-mutated non-small cell lung cancer by 44%.

Yoni Tyberg, associate director of the Special Program Staff in the Office of New Drugs, provided an update on efforts to modernize CDER processes.

Diversity Action Plans are now required to be submitted by medical product sponsors after changes governed by the Food and Drug Omnibus Reform Act.

This alert follows a similar one from the European Medicines Agency in October 2023 amid a rise in demand for the diabetes medication that, in turn, created a shortage.

The guidance document provides information about the agency’s intentions for assigning a goal date for a facility’s inspection readiness under GDUFA.

Imfinzi (durvalumab) combined with chemotherapy decreased the risk of disease progression or death by 58% in a 700-patient trial.

CDER's new program will create discussion around AI and be administered through the Emerging Drug Safety Technology Program.

Breyanzi is made from the patient’s own T cells, which are genetically reengineered into CAR-T cells and then delivered through infusion as a one-time treatment.