pharmaphorum

FEBRUARY 2, 2022

Pharming has taken a step closer to getting a second product to market, after its rare disease therapy leniolisib met its objectives in a pivotal trial, setting up regulatory filings in the coming months. As it stands, the only other candidate for the disease listed in the clinicatrials.gov database is generic drug rapamycin.

Trials 52

Trials Drugs Sales Generic Drugs 52

The Pharma Data

MAY 6, 2021

CSL closes deal for UniQure gene therapy in hopeful sign for M&A ( BioPharmaDive ). Dyno raises $100 million to bring artificial intelligence to gene therapy ( STAT ). Biogen’s Alzheimer’s drug may only be cost effective at $2,500 a year ( STAT ). OcuWho? .

Sales 52

Sales Vaccination Vaccine Gene Therapy 52

XTalks

AUGUST 2, 2023

But all three drugs have new competitors. Meanwhile, BioMarin’s gene therapy Roctavian is approved by the FDA for the treatment of hemophilia A. Additionally, Bayer has formed a partnership with Mammoth Biosciences to broaden its product portfolio with innovative gene-editing technology. Both are anti-CD20 agents.

Sales 98

Sales Manufacturing FDA Approval Life Science 98