Regulatory Oversight and Compliance

A Phase III trial demonstrated mNEXSPIKE’s non-inferior efficacy compared with Moderna’s original COVID vaccine, Spikevax.

The concerns of industry experts expressed at INTERPHEX in April 2025 are still pertinent in an uncertain global political climate.

Bempikibart (ADX-914) is a human anti-IL-7Rα antibody that blocks the IL-7 and TSLP pathways, which have been implicated in driving T cell-mediated pathological processes in autoimmune diseases.

The company said that Vyvgart (efgartigimod alfa) is the first novel mechanism of action for CIDP treatment in more than 30 years.

The policy announced by Commissioner Martin A. Makary, MD, will not preclude employees of regulated companies from presenting their views to committees, and rare exceptions will be allowed.

The recombinant vaccine, under the brand name Vimkunya, is recommended for persons age 12 and older who are traveling to a country or territory where there is a chikungunya outbreak.

Vitrakvi (larotrectinib) was first granted accelerated approval by FDA in November 2018.

Development of the state-of-the-art facility was made possible by a £2 million (US$2.6 million) round of funding, and a successful inspection by MHRA was completed in March 2025.

3D printing of personalized medications is currently possible under existing compounding regulations, offering enhanced process control through automation. But new legislation coming in 2025 will allow 3D printing as part of a distributed manufacturing framework.

The authors examine the risks of extractables and leachables, and present solutions that emphasize the importance of a strategic, multi-prong approach.

Three of the five are biosimilars, and seven additional medicines received recommendations for indication extensions.

In the latest of a series of warning letters to India-based API manufacturers, FDA issued a warning letter to Aspen for deviations in CGMP in the production of APIs.

The company’s state-of-the-art sterile drug product manufacturing site in Saint-Beauzire, France, opened in February 2023.

Fabhalta (iptacopan) received a positive opinion for treatment of C3G from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) and has been approved twice before by FDA.

The EU medicines agencies’ network strategy, EMANS, is an update of the five-year strategy previously developed to cover the period 2021 to 2025.

Rivaroxaban, long known under the brand name Xarelto, is an anticoagulant, a classification that is among the most-prescribed medications in the United States.

The alterations are the result of an agency review of findings from a clinical trial, and data from required postmarket studies.

CHMP gave its opinion and a recommendation for marketing authorization to Fabhalta (iptacopan), an oral, Factor B inhibitor of the alternative complement pathway.

More than 6000 rare diseases—those which affect fewer than one in 2000 people each—are acknowledged in the medical community, with 72% of them being genetic.

Treatments for autoimmune diseases, multiple cancers, and skin blistering have been recommended for marketing approval.

Cerebrotendinous xanthomatosis, or CTX, is a metabolic disorder caused by a gene mutation that results in a deficiency of an important enzyme in the ability of the human body to break down fats.

Seladelpar, which is marketed in the US as Livdelzi, was recommended for an orphan drug designation by CHMP in December 2024.

Tenosynovial giant cell tumor (TGCT) is rare and non-malignant, forming in or near joints, but which if left untreated can cause damage to joints and surrounding tissue, potentially leading to significant disability.

The agency has received certification with the Eco-Management and Audit Scheme for its commitment to environmental sustainability.

The approval makes Evrysdi the first and only tablet for treating spinal muscular atrophy.

With the approval, FDA granted SpringWorks a rare pediatric disease priority review voucher.

Acoramidis was approved by FDA in November 2024 and is prescribed in the US under the brand name Attruby.

While this approval addresses breast cancer, olaparib in combination with durvalumab was approved by the EU in August 2024 for treatment of endometrial cancer.

These infections can be caused by certain Gram-negative microorganisms, making the infections challenging to control due to high antimicrobial resistance.

EMA introduced its New Fee Regulation in January 2025, but what impact will the new fees and charges have on medicine developers?