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The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Medannex's novel therapy MDX-124, for the treatment of biliary tract cancer (BTC). The FDA’s decision is based on emerging clinical data from the ongoing first-in-human Phase 1b ATTAINMENT clinical trial, which shows promising efficacy signals in patients treated with MDX-124 monotherapy. Durable disease control and tumour shrinkage have been observed in several BTC patients, with no significant side effects. 

This is the first-ever autoimmunity study to evaluate a potential functional cure that targets the antigenic source – the origin of autoimmune disease.

First non-hormonal menopause treatment approved for use in the UK by the MHRA, with early-stage development at Stevenage Bioscience Catalyst

Microbiotica announces that patient recruitment is complete in its Phase 1b First-in-Human trial, COMPOSER-1, of MB310 in ulcerative colitis (UC) patients.

Rinri Therapeutics is focused on transforming sensorineural hearing loss (SNHL) with regenerative cell therapy, providing new therapeutic options for the treatment of this major global unmet medical need.

A Scottish clinical development company is redefining early stage development, taking years off the time it takes for new life improving treatments to become available to patients. BDD Pharma Ltd – based at BioCity Glasgow, one of two Pioneer Group sites in Scotland – has also developed its own, in-house technologies. One of them - OralogiK™ - is a unique patented drug delivery tablet system that enables drugs to be released inside the body at the optimum time, hours after taking the tablets.

In this blog Richard Turner, the new vice chair of FTAC, examines the UK's overhauled R&D tax regime, which provides competitive incentives and a stable fiscal framework.

WHO members adopted the pandemic preparedness accord as an international treaty at the World Health Assembly. In this blog, Senior Policy and Public Affairs Manager Linda Bedenik looks at what the agreement means for life sciences.

Poolbeg Pharma announces that the US Food and Drug Administration has granted Orphan Drug Designation to POLB 001 as an oral preventative therapy for T-cell engager bispecific antibody-induced Cytokine Release Syndrome.

Ferrer enters into a collaboration and license agreement with Prilenia for the commercialization and co-development of Pridopidine

MHRA has granted conditional marketing authorisation for Autolus' treatment of adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-ALL).

European Medicines Agency (EMA) has granted orphan drug designation to PS-002, Purespring’s lead programme, in IgA nephropathy (IgAN).

Sarah Brockbank, Strategy Leader – Complex Medicines at Medicines Discovery Catapult, shares insight into a new regulatory roadmap created by the UK’s Intracellular Drug Delivery Centre (IDDC).