Ad Header

PharmaLive

Slogan

The Pulse of the Pharmaceutical Industry

  • Filter By Category

  • Filter By Author

Orchard Therapeutics Licenses Potentially Life-Changing MPS-I Gene Therapy Program

Orchard Therapeutics secured an exclusive license for an ex vivo autologous hematopoietic stem cell gene therapy program for the treatment of MPS-I developed by SR-Tiget.

Read More »

Novartis’ SMA Gene Therapy Treatment Approved by FDA, Priced at $2.125 Million

The U.S. Food and Drug Administration approved Zolgensma, a gene therapy that Novartis called a life-changing medicine, for children younger than 2 years of age who have been diagnosed with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 gene.

Read More »

Waylivra Approved In EU For Treating FCS

Akcea Therapeutics Inc. and Ionis Pharmaceuticals Inc. announced that Waylivra received conditional marketing authorization from the European Commission as the only therapy for FCS.

Read More »

Second death in Novartis gene therapy trials under investigation

Novartis AG, which previously announced positive interim trial results for the company’s experimental gene therapy for spinal muscular atrophy, said an investigation is underway into whether a second trial death could be related to the treatment.

Read More »

Regeneron forays into gene-silencing therapies with Alnylam tie-up

Regeneron Pharmaceuticals is investing $800 million in Alnylam Pharmaceuticals to develop treatments for eye and central nervous system diseases using gene-silencing RNA interference technology.

Read More »

Biogen SMA drug price, Novartis estimates for its treatment far too high – U.S. group

Biogen Inc. should slash the price of the spinal muscular atrophy (SMA) drug Spinraza, and the $4 million to $5 million Novartis has said the company’s experimental gene therapy Zolgensma for the disease is worth is excessive, an independent U.S. organization that reviews the value of medical treatments said.

Read More »

FDA Approves ADMA’s Asceniv for Immune Deficiency

The U.S. Food and Drug Administration approved ADMA Biologics’ Asceniv to treat primary humoral immunodeficiency disease (PIDD or PI) in adults and adolescents.

Read More »

EU panel approves drug for rare genetic disease

An advisory committee of the EMA recommended conditionally approving a drug from Akcea Therapeutics and Ionis Pharmaceuticals that aims to treat a rare genetic disease.

Read More »

Roche acquires Spark Therapeutics for $4.3 billion

Roche is buying U.S.-based gene therapy specialist Spark Therapeutics for $4.3 billion after developments in this area convinced the Swiss drugmaker to “step up”, Chief Executive Severin Schwan said.

Read More »

7 Biotech Trends to Look Out for in 2019

Here are seven trends most likely to be hitting the news cycle in 2019 according to BioSpace.

Read More »

Ad Right Top

MedAdNews

Extensive pharmaceutical business and marketing intelligence. For back issues, please contact MDAD@kmpsgroup.com.

June 2019 Focus: Payer access, biotech/biopharma, DTC, rare diseases, and more!

Subscribe

Ad Right Bottom