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ESMO 2017 — Part 2: Building on Biomarkers

The news from ESMO in September once again has highlighted the utility and need for biomarkers to help determine not only who will likely benefit from a particular immuno-oncology (IO) treatment, but also who may require a combination of such therapies right from the start.   While higher levels of PD-L1 have generally correlated with...

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...

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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...

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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...

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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...

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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...

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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...

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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...