17
Sep
2018
Tag: rare diseases
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...
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...
2
Mar
2017
Bionest
Chan Zuckerberg Initiative Makes First Bio Research Awards
Last fall, the Chan Zuckerberg Initiative announced plans to invest $3 billion over 10 years in scientific efforts with an ambitious long-range aim to “cure, prevent or manage” all diseases. As a first part of that effort, they directed $600 million towards the establishment of the CZ Biohub in San Francisco’s Mission Bay neighborhood. This...
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....
6
Apr
2016
Bionest
“Beyond the Pill” — Part Two: Value-Added Services Offer Benefit to Multiple Audiences
In our first “Beyond the Pill” post, we discussed the increasing move towards patient-centric programs that go past an actual prescription to help build relationships with patients and physicians and facilitate the provision of a number of related services to improve compliance and treatment outcomes and keep patients on drug long-term. The benefit of such...
23
Mar
2016
Bionest
Launch or License: Some Points to Consider, When You’re Considering Your First European Launch
For many young biotech and pharma companies, the decision about how to take their first drug to Europe has been simple: just license the European marketing rights to a company that already is well established there, and collect milestone payments and a stream of royalties. For an inexperienced company, dealing with the European regulatory and...
8
Mar
2016