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… Read »

Liquid Biopsy – Looking Beyond EGFR

In April, two important reports were published — one in the New England Journal of Medicine, one in Nature — that added evidence in support of liquid biopsy as a means of monitoring for the recurrence of cancer and to… Read »

ASCO 2017 — Some Take-Aways

Several members of the Bionest team attended ASCO this year — the biggest cancer conference in the world, attracting over 30,000 oncology researchers, biopharmaceutical executives, public health experts, investors and journalists.   As we predicted in our pre-ASCO post, one… Read »

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… Read »

Roche Gains Important MS Approval — and Takes Unprecedented Pricing Steps

On March 29, the US Food and Drug Administration approved Roche’s Ocrevus (ocrelizumab) for the treatment of either relapsing multiple sclerosis (MS), or primary progressive MS (PPMS).  The approval was particularly noteworthy as Ocrevus is the first therapeutic approved for… Read »

FDA Changes the Game: First Cancer Drug Approval Based on Biomarker

The U.S. Food and Drug Administration has issued its first drug approval to a treatment for patients with a specific genetic biomarker, rather than based on an initial tumor location, such as breast cancer. On May 23, the agency granted… Read »

It’s ASCO Time Once Again!

ASCO is the largest annual gathering of the worldwide oncology community, and this year’s event takes place this coming weekend, from June 2-6 in Chicago, Illinois.  About 10 days ago, the association released its program and a wide slate of… Read »

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… Read »

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… Read »

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… Read »

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