Geography

India is pressing ahead with “big shifts” in its regulatory approach for cell and gene therapies. Reduced “layers of review”, tighter internal timelines, upcoming guidelines to facilitate CGT development and reforms in the constitution of expert committees are some of the key changes.

After almost seven years, China’s NMPA has once again published draft measures for clinical trial data protection, opening these up to public comments. While specific protection periods by drug category are proposed, some clarifications are still needed.

Japan's trade ministry is providing new support to build domestic capacity for new modalities including cell and gene therapies, as part of wider efforts to support the national bioventure ecosystem.

US-based Colossal Labs has claimed to bring back the dire wolf from extinction. While the science is not as dramatic in India yet, cell and gene therapies are making progress as treatments for cancer and other diseases. Pink Sheet takes a look at what is driving CGT success and growth.

Efforts by the medicines regulator, the MHRA, to clear all backlogs relating to its statutory functions and maintaining predictable review timeframes have put pressure on its staff.

Pharmaceutical companies need to “pressure test” their clinical development strategies early for health technology assessment purposes, particularly in light of the new EU HTA Regulation, a life sciences consultant says.

This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.

The European Medicines Agency has recommended 16 drugs for EU-wide approval, including Vertex’s cystic fibrosis drug Alyftrek and five other orphans.

The Pink Sheet highlights recent comments and insights from pharma officials and executives on key issues we are covering.

Mechanisms in the draft treaty that the more than 190 member states of the World Health Organization have finally agreed to are expected to “materially affect companies,” particularly those that develop, manufacture or distribute pandemic-related health care products.

The International Council for Harmonisation has consolidated and modernized its existing stability guidelines into one document, and addressed modern stability testing approaches like modeling, bracketing, and matrixing.

While the biosimilars industry has welcomed individual pockets of progress around regulatory streamlining, it is essential that approaches from global authorities move forward together if they are to have a meaningful impact on biosimilar development. At Medicines for Europe’s annual biosimilars conference, regulators from around the world talked about how their thinking is converging.

CMED, which sets medicine prices in Brazil, has published the maximum price adjustment index for 2025.

The Brazilian medicines regulator will also offer more clarity on the requirements for radiopharmaceuticals that are exempt from registration.

Brazil’s drug regulator ANVISA has also published its first post-marketing authorization monitoring reports on advanced therapies Yescarta, Zolgenma, Kymriah, Carvykti and Luxturna.

Health technology assessment bodies in England, the US, Canada, Australia, the Netherlands and Colombia have joined forces to produce guidance for drug developers on using surrogate endpoints for cost-effectiveness analyses.

The African Medicines Agency is to appoint a director general and become operational at the end of this month.

Israel has introduced a new framework to expedite the marketing approval of medicines, make the country a more attractive destination for drug registration and help reduce drug prices through market competition.

In a historic move for Africa, five products have been backed for approval via a pilot that tested a continental listing process.

Efforts have stepped up to enhance Africa’s capacity to meet international good manufacturing practice standards, empower regulators across the continent, and drive sustainable growth in the pharmaceutical sector.

In an unusual move, Tracy Beth Høeg, a special assistant to FDA Commissioner Martin Makary, is leading the continued negotiations on Novavax’s delayed COVID-19 vaccine approval.

Respondents to a Senate investigation that could re-energize 340B reform efforts said hospitals offer patient assistance programs and use 340B revenue for “capital improvement projects” and “community benefit programs,” though they do not account for specific program revenue allocations.

Agency staff repeatedly said the BLA based on a single-arm study in 55 patients lacked substantial evidence of effectiveness in steroid-refractory acute graft-versus-host disease and a randomized trial was needed, but changed course “based on additional consideration” after a second CRL.

The Pink Sheet’s Drug Review Profile looks at the timeline for the clinical development and US FDA review of Mesoblast’s remestemcel for graft-versus-host disease.