Regulatory Oversight and Compliance

Two drugs, Miplyffa (arimoclomol) and Aqneursa (levacetylleucine), have been approved by FDA to treat neurological symptoms associated with Niemann-Pick disease, type C in adults and children.

FDA’s Darby Kozak provided commentary on the anniversary of the Drug Price Competition and Patent Term Restoration Act of 1984.

The approval of EBGLYSS (lebrikizumab-lbkz) by FDA puts a new biologic treatment for moderate-to-severe atopic dermatitis on the market for patients whose symptoms are not well controlled with topical treatments.

Approvals were recommended for treatments of ovarian cancer, lung cancer, bleeding disorders, respiratory disease, and more.

The company said this new commitment to its facility in Germany goes hand in hand with its recently announced ReciPredict, an initiative intended to streamline the product development cycle.

Bio/pharma companies should be approaching their decarbonization journey as a strategic business move for the future as industry stakeholders are placing greater value on sustainability.

What impact will the AI Act have on pharma and digital medicine?

Phase III trial results showed that Imfinzi (durvalumab in combination with chemotherapy reduced the risk of recurrence, progression, or death by 32% in patients with resectable non-small cell lung cancer before and after surgery.

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.

This Behind the Headlines news roundup panel discussion covers news items such as the resurgence of mpox in Africa, “medical tourism” in India, Eli Lilly’s new Boston CGT facilities, and contract development and manufacturing organization (CDMO) projected growth rates for the 2023–2029 period, and the reasons behind them.

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.

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

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

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 epinephrine nasal spray was approved for the emergency treatment of type 1 allergic reactions in adults and children.

The program, for which visits will be scheduled through September 2025, does not replace or supplement a regulatory inspection.

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

Germany’s Medical Research Act allows pharmaceutical companies to keep drug prices confidential.