Therapeutic Areas in Focus

Volume 76 Spring 2024

Dive into the latest pharma news from Pfizer’s Beqvez gaining FDA approval for haemophilia B treatment (page 6) and Johnson & Johnson’s Rybrevant gaining a positive opinion from the Committee for Medicinal Products for Human use for lung cancer treatment (page 6), to the FDA reviewing GSK’s meningococcal vaccine (page 8) and ANGLE and AstraZeneca signing a commercial agreement (page 9).

This issue’s rare disease section is bursting with articles spanning a range of topics within the sector. First up, Quotient Sciences consider orphan drug development and CRO/CDMO collaboration, specifically in paediatric rare diseases (page 10), while ProPharma explores clinical trials for rare diseases and how these could be improved (page 14). Alexion then tells us about the challenges facing rare disease treatment and the importance of patient-centricity (page 20), and Veeva assesses how companies are overcoming challenges related to rare disease education, development and launch of new medicines (page 22). Finally, Synaptix Bio tells us about the problems facing the treatment of rare diseases (page 17).

Psychoactive medicine is a huge area of development within the pharma industry, as various companies look for alternative options to traditional standard-of-care treatments for many conditions – including, but not limited to, mental health conditions. In this issue, Compass Pathways explores the need for new treatments in the mental health space, as well as assessing whether psychoactives could be the way forward (page 24). Also in our psychoactives section, Beckley Psytech tells Pharmafile about the use of psychedelic medicine for the treatment of psychiatric and neurological conditions, and how this field could develop in the future (page 26).

This issue also covers all aspects of the fascinating field of women’s health and the developments being made in the sector. To open the section, Organon explores the impact of innovations in contraception and how this can reduce unintended pregnancy rates (page 30). Then, InnoScot Health assesses the gender health gap before considering the way forward in closing this and improving women’s healthcare overall (page 32).