Metabolic Disorders

Ultra-Rare Disease Drug Loargys Wins English Funding Ahead Of US FDA Verdict

Immedica’s Loargys is set to become the first disease modifying treatment for arginase 1 deficiency to be reimbursed in England. Meanwhile, the US regulator is reviewing a resubmitted marketing application for the product and has set a target action date of Feb. 23.

Chinese ADC, Cell/Gene Therapy Biotechs Ride Funding Spree

2026 has already seen several major new funding rounds for Chinese biopharma ventures, including a $70m financing for CAR-T specialist Oricell and a $40m Angel round for ADC specialist Tosun.

England Finalizes Access For SGLT-2 & GLP-1s In Diabetes, Unlocks £560m Savings

SGLT-2 inhibitors should be used to treat patients with type 2 diabetes much earlier in their treatment, the English health technology assessment institute said, adding that using generic dapagliflozin would save the National Health Service £560m.

AstraZeneca Bets $18.5bn On CSPC’s Sustained-Release And AI Peptide Platforms In Weight Management

UK major will bag CSPC’s eight preclinical drug candidates in weight management, including most advanced asset SYH2082, a long-acting GLP-1/GIP receptor agonist with once-monthly dosing potential.

OrsoBio To Advance TLC-6740 After Successful Obesity Combo Trial

The California firm announced positive data from its early-stage randomized study of TLC-6740 combined with Lilly’s tirzepatide in patients with obesity.

HighTide’s First-In-Class Diabetes Contender Numerically Superior To Farxiga Glycemic Control

In the head-to-head Phase III HARMONY trial in metformin-failed type 2 diabetes, HighTide's HTD1801 yielded a mean 1.12% reduction in HbA1c from baseline at 24 weeks, versus 0.93% for AstraZeneca’s Farxiga.

Wiskott-Aldrich Syndrome Gene Therapy Among 10 New Drugs To Win EMA OK

While the European Medicines Agency’s human medicines committee voted in favor of EU approval for 10 new products, it reaffirmed its previous decision not to grant Aqneursa new active substance status.

Novel Alzheimer’s Drug Blarcamesine Scheduled For High-Stakes EMA Oral Explanation

The EMA has scheduled oral explanation meetings this week for Anavex’s blarcamesine and three other products that are nearing the end of the regulatory review cycle. These meetings usually represent the final chance for sponsors to persuade the agency that their product merits approval.

Topline Phase III Efpeglenatide Data Raise Hopes For Hanmi’s Obesity Contender

Hanmi is planning to file for Korean approval of the country's first homegrown obesity contender efpeglenatide by year-end, following positive topline Phase III data for the Sanofi U-turned asset.

Sanofi’s Rilzabrutinib & Belumosudil Among Seven Drugs Facing EMA Oral Explanations

The sponsors of seven new medicines nearing the end of their EU regulatory review are due – or possibly due – to appear before the European Medicines Agency’s human medicines committee, the CHMP, to address remaining concerns about their marketing applications.

First-In-Class Bronchiectasis Treatment Among 14 Drugs Awaiting EMA Verdict

The European Medicines Agency is this week expected to recommend whether EU marketing approval should be granted to myriad new products, including therapies for bronchiectasis, RSV, menopause-related symptoms and type 2 diabetes.

First-Of-A-Kind Nasal Spray For Pain In Children Among 13 New EU Filings

Cessatech and Proveca’s investigational, sufentanil/ketamine fixed-dose combination analgesic nasal spray is among the latest drugs that the European Medicines Agency has started to review for potential pan-EU marketing authorization.