18
Feb
2019
Tag: orphan drugs
29
Oct
2018
Bionest
First RNAi Drug Hits the Market — At a Very High Price
On August 13, the U.S. Food and Drug Administration (FDA) approved Alnylam’s Onpattro (patisiran), rapidly followed by that product’s approval in Europe three weeks later. Onpattro, which we previously wrote about, is the first RNAi-based treatment to reach the marketplace. It is intended to treat peripheral nerve disease caused by a rare genetic condition called...
15
Oct
2018
Bionest
Gene Therapy Update Part 2
Last week we wrote about recent news in the field of CRISPR/Cas9 research. But CRISPR is not the only approach to gene editing under development, nor the most advanced. First Human Gene Editing Data — But Not from CRISPR Sangamo Therapeutics, Inc. has been developing an approach to gene editing based on zinc...
17
Jul
2017
Bionest
Gene Therapy: Coming of Age Perhaps, but Who Will Pay?
New technologies tend to go through cycles of interest and attention. Initial excitement about the potential of a technological advance too often builds to over-blown hype, followed by a quick reversal of interest in the aftermath of a big disappointment. But if continued research later produces truly encouraging results, interest can build again as time...
23
May
2017
Bionest
And They’re Off….. Watching the CAR-T Race
On March 29, Novartis announced that the US Food and Drug Administration had accepted the company’s BLA filings for CTL019 (tisagenlecleucel-T) for the treatment of pediatric and young adult patients with relapsed and refractory B-cell acute lymphoblastic leukemia (ALL). Two days later, Kite Pharma announced that they had completed the rolling submission for their own...
16
May
2017
Bionest
A Look at Duchenne Muscular Dystrophy: Part 3 – The Increasing Activism and Influence of Patient Advocacy Groups
On September 19, 2016 the US Food and Drug Administration approved Sarepta’s Exondys 51 (eteplirsen) injection, the first drug approved to treat the underlying cause of Duchenne muscular dystrophy (DMD). Exondys 51 is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13...
8
May
2017
Bionest
A Look at Duchenne Muscular Dystrophy: Part 2 – About DMD, and Approaches to Treatment, Old and New
Duchenne Muscular Dystrophy (DMD) is a rare genetic disease characterized by progressive muscle degeneration and weakness. It is one of the most frequent genetic disorders, affecting approximately 1 of 3,600 (primarily) male children. The disease is caused by a mutation in the gene for dystrophin on the X chromosome that results in the absence of...
2
May
2017
Bionest
A Look at Duchenne Muscular Dystrophy: Part 1 – Marathon Pharmaceuticals, PTC Therapeutics, and Drug Pricing
In February of this year, Marathon Pharmaceuticals received approval for Emflaza (deflazacort) tablets and oral suspension for the treatment of patients over the age of five years with Duchenne muscular dystrophy (DMD), regardless of the particular genetic mutation associated with their condition. The drug — an older corticosteroid with no patent protection which has...
30
Jan
2017
Bionest
The Orphan Drug Act: Successes and Challenges
By Alain J. Gilbert, Co-Chair, Bionest Partners Rare and orphan diseases have grown dramatically as a focus of drug development since the passage of the Orphan Drug Act and the institution of other regulatory and financial incentives in both the United States and Europe aimed at spurring development for such conditions. Franҫois Beaubien, Ph.D....
14
Sep
2016