Drug Development

FDA approves leucovorin for cerebral folate deficiency, signaling how literature-based evidence can support rare disease approvals without clinical trials.

The industry is shifting toward increasingly complex, precision-driven modalities for the treatment of cancer.

The 2mg/kg every-4-weeks dosing regimen for pegunigalsidase alfa in adults with Fabry disease who are stable with an enzyme replacement therapy has been approved in Europe.

Developments across the sector highlight a period of rapid acceleration and structural modernization.

Teclistamab plus daratumumab gains FDA approval for relapsed/refractory multiple myeloma, showing 83% reduction in disease progression versus standard care.

In part four of this four-part series on rare diseases, the authors examine how orphan drug economics are reshaping biopharma deal-making behavior, accelerating earlier-stage transactions, and shifting the geography of innovation sourcing, with future rare disease innovation increasingly emerging outside traditional US and European development centers.

Elisabeth Gardiner, PhD, CSO of Tevard Biosciences, explains some of the challenges associated with the development of treatments for rare diseases.

Trust-based CDMO partnerships are becoming essential to accelerate injectable drug development, manage risk, and ensure resilient biopharma supply.

Industry experts discuss the benefits of utilizing contract development and contract research organizations in drug discovery and development.

In this episode of Ask the Expert, Susan J. Schniepp, Nelson Labs, and Siegfried Schmitt, Parexel, discuss the benefits of orphan drug development and how a mid-sized company can work with regulators through the pathway to approval.

In part three of this four-part series regarding rare diseases, the authors examine how revenue concentration and accelerating patent cliffs undermine the traditional blockbuster model, whereas orphan drugs offer a structurally different strategy for stabilizing biopharma portfolios through more durable, predictable, and risk-adjusted long-term value creation.

Suzanne Jansen, MSc, PharmD, ICON, says early-phase clinical success depends on flexible, simple formulations that support wide dose ranges, matching placebos, and robust stability.

New disease-modifying agents are being evaluated in the clinic that could change the course of treatment for Alzheimer’s disease patients.

PharmTech spoke with women working in the pharmaceutical industry about the innovations they are seeing in bio/pharma and the role women play in the development of critical treatments for patients.

PharmTech spoke with Klaudia Lechowska, Business Development Specialist at Mabion Biologics CDMO, about how women working in STEM fields bring unique perspectives to the important work of rare disease treatment.

Integrated CDMO networks streamline complex drug development across advanced modalities like ADCs.

In part two of this four-part series regarding rare diseases, the author examines how economic and structural forces are driving an access gap.

FDA has granted priority review to AstraZeneca's Datroway for the first-line treatment of metastatic TNBC treatment.

PharmTech spoke with Dr. Stacy Lindborg, president and CEO of IMUNON, about her experience at IMUNON, which is developing a novel DNA-based approach to treat ovarian cancer.

The companies’ “Change the Target. Change What’s Possible” targets the potential of Factor XIa inhibition in the development of treatments for thromboembolic disease.

PharmTech spoke with Katy MacLellan, Technical Team leader, Symbiosis Pharmaceutical Services Ltd., about the women in the pharmaceutical industry that inspire her.

Repertoire’s T cell receptor-epitope discovery platform will be used to develop tolerizing therapy development candidates with Eli Lilly and Company.

In part one of this four-part series regarding rare diseases, the author explores how rare disease treatment development evolved from a niche area to a cornerstone of biopharma growth.

Visit PharmTech.com for upcoming coverage of the International Day of Women and Girls in Science on February 11.

The European Commission has approved GSK’s adjuvanted recombinant respiratory syncytial virus vaccine, Arexvy, for use in adults aged 18 years and older.