Pharma/Biotech – USA, Europe, Asia and ROW Regulatory News – Feb 2025

USA

FDA Approves First Rapid-Acting Insulin Biosimilar Product for Treatment of Diabetes

The U.S. Food and Drug Administration approved Merilog (insulin-aspart-szjj) as biosimilar to Novolog (insulin aspart) for the improvement of glycemic control in adults and pediatric patients with diabetes mellitus. Merilog, a rapid-acting human insulin analog, is the first rapid-acting insulin biosimilar product approved by the FDA. As a rapid-acting insulin, Merilog helps to lower mealtime blood sugar spikes to improve control of blood sugar in people with diabetes. The approval is for both a 3 milliliter (mL) single-patient-use prefilled pen and a 10 milliliter (mL) multiple-dose vial.

FDA Approves First Treatment for Cerebrotendinous Xanthomatosis, a Rare Lipid Storage Disease

The U.S. Food and Drug Administration approved Ctexli (chenodiol) for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Ctexli is the first FDA-approved drug to treat CTX, a very rare lipid storage disease.

The FDA is dedicated to supporting new drug development for rare diseases including very rare metabolic diseases like cerebrotendinous xanthomatosis,” said Janet Maynard, M.D., M.H.S., director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, in the FDA’s Center for Drug Evaluation and Research. “CTX is a progressive multisystemic disorder that significantly impacts patients and previously lacked approved treatments. Today’s approval provides a safe and effective treatment option for CTX.

EUROPE

Combination of cystic fibrosis medicines to treat patients with rare mutations

EMA’s human medicines committee (CHMP) has recommended extending the therapeutic indication of two medicines, Kaftrio (ivacaftor / tezacaftor / elexacaftor) and Kalydeco (ivacaftor) for the treatment of cystic fibrosis, to include their use in combination for patients aged two years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

First topical gene therapy treatment for dystrophic epidermolysis bullosa

EMA has recommended granting a marketing authorisation in the European Union (EU) for Vyjuvek (beremagene geperpavec) to treat wounds in patients of all ages with dystrophic epidermolysis bullosa (DEB).

Dystrophic epidermolysis bullosa is a serious, ultra-rare genetic blistering disease caused by mutations in the collagen type VII alpha 1 chain (COL7A1) gene. This gene is involved in the production of type VII collagen (COL7), a protein crucial in supporting and structuring the layers of the skin. DEB is characterised by skin fragility, blister formation, small white bumps (milia) and scarring, and is usually present at birth. Blisters and erosions may also develop in the mouth, oesophagus, rectum, genitourinary system and eyes, or other mucous tissue. Healing of blisters and erosions can result in debilitating scarring and other serious conditions. Recessive DEB, the most severe form of DEB, can lead to the loss of fingernails and toenails, joint deformities, vision loss and unusually aggressive squamous cell carcinoma (a type of skin cancer).

AUSTRALIA

Nitrosamine and Nitroso-structure impurities acceptable intakes update

TGA has published updated information for nitrosamine impurities and other nitroso-structure impurities in medicines consistent with recent EMA updated information- external site. The changes include additional clarification for sponsors and manufacturers of the TGA’s expectations, minor editorial amendments, increases to the AI limit for some impurities and inclusion of recently internationally determined AI limits for numerous nitrosamine impurities and other nitroso-structure impurities in medicines. The AI changes are tabulated on the updates to nitrosamines and other nitroso-structures page.

BRAZIL

RDC 954/2024 – Understanding Key Points and Impacts on Drug Registration

Anvisa will hold an in-person event entitled “RDC 954/2024 – Understanding key points and impacts on drug registration”. The meeting will be an opportunity to clarify doubts and deepen understanding of the regulatory changes introduced by Collegiate Board Resolution (RDC) 954/2024.

CANADA

Government of Canada takes further action to address fentanyl precursor chemicals

Health Canada accelerating Canada’s work to detect, disrupt, and dismantle the fentanyl trade. Illegal drugs are having a devastating impact on people and communities, including creating security challenges associated with their illegal production, importation, trafficking, and related crimes. Health Canada plays a critical role in supporting Canadian law and border enforcement in their activities to disrupt the global drug threat and is taking concrete action to keep communities safe on both sides of the border.

JAPAN

PMDA-ATC Pediatric Review Seminar 2025

The Pharmaceuticals and Medical Devices Agency (PMDA) is pleased to announce the “PMDA-ATC Pediatric Review Seminar 2025” for officials of new drug application reviewers from overseas regulatory authorities. The seminar will be held IN PERSON at the PMDA Office in Tokyo from June 9 to 12, 2025

SINGAPORE

HSA Seized Over 970,000 Units of Illegal Health Products and Removed More Than 7,000 Illegal Product Listings in 2024

The Health Sciences Authority (HSA) has continued to step up ground and online surveillance efforts to curb the sale and supply of illegal health products in the past year. Coordinated efforts involving targeted operations, intelligence sharing, and joint enforcement with both local and overseas agencies led to the seizure of 970,707 units of illegal health products and the removal of 7,351 listings from local e-commerce and social media platforms. This multi-pronged approach has disrupted the supply of illegal health products and protected the public from potential harm posed by these products.

UK

mRESVIA RSV vaccine approved to protect patients aged 60 and over

The Medicines and Healthcare products Regulatory Agency (MHRA) has approved an mRNA respiratory syncytial virus (RSV) vaccine (brand name mRESVIA) to protect patients aged 60 and over against lower respiratory tract disease caused by RSV.

RSV is a common virus that spreads very easily and causes respiratory tract disease in people of all ages. RSV infection can be mild, with cold-like symptoms including blocked nose, cough, and/or sore throat. However, the virus can also cause more serious problems, such as lung infections and pneumonia. Older adults are at risk of more serious complications that can lead to hospital admission and even death.

Nemolizumab approved to treat prurigo nodularis and atopic dermatitis (eczema) for patients in the UK

The Medicines and Healthcare products Regulatory Agency (MHRA) has approved the medicine nemolizumab (brand name Nemluvio) for the treatment of two skins conditions – moderate to severe prurigo nodularis for adults aged 18 and above, and moderate-to-severe atopic dermatitis (eczema) for adults and adolescents aged 12 and above. 

Prurigo nodularis is a chronic skin condition that causes hard, itchy bumps called nodules.  The safety and efficacy of nemolizumab for this condition were demonstrated in two clinical trials in adults (aged 18 yrs and over). The safety and efficacy of nemolizumab have not been established in patients below the age of 18 years with prurigo nodularis.