17
Oct
2017
Tag: competitive differentiation
5
Jul
2017
Bionest
FDA: Thinking “Outside the Box”
On May 17, the U.S. Food and Drug Administration granted an expanded approval to Vertex’s Kalydeco (ivacaflor), tripling the number of mutations in the cystic fibrosis (CF)-associated gene CFTR that the drug can treat, from 10 to 33. The agency’s action was unusual in that, for the first time ever, they based their decision on...
14
Jun
2017
Bionest
Towards a Universal Immuno-oncology Assay Predictive of Drug Response
We have written frequently about the growing importance of biomarkers and how diagnostics will increasingly play a dominant role in the way that therapeutics are prescribed and reimbursed. Immuno-oncology (IO) treatments in particular are expected to benefit from the availability of truly predictive biomarkers, based on the dramatic treatment gains they have achieved for a...
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...
17
Apr
2017
Bionest
Reflecting on AACR
The American Association for Cancer Research annual meeting is always a source of major news and discussion on the oncology research front, and the 2017 meeting held earlier this month was no exception. One area of particular interest was the encouraging data on long-term survival that is emerging for some patients treated with immuno-oncology...
5
Apr
2017
Bionest
PARP Inhibitors Chalk Up Impressive Gains in Ovarian Cancer Maintenance Therapy
On March 27, the FDA gave Tesaro a broad approval for its PARP inhibitor, niraparib, as maintenance therapy for patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer after a complete or partial response to platinum-based chemotherapy. The FDA decision came well in advance of the PDUFA date set for June 30, and...
21
Mar
2017