Here are a few of the stories that grabbed our attention in December 2017:
Bloomberg reported that only five patients to date have received Gilead’s Yescarta since its FDA approval in October, while more than 200 are on waiting lists to receive the breakthrough CAR-T therapy. Some believe the holdup may be due to reimbursement challenges that new CAR-T therapies currently present to Medicare and Medicaid. The delays in payment are forcing hospitals to choose between covering the cost themselves or asking patients in need to wait for the therapy.
Stat Plus kicked off awards season by naming Juno Therapeutics “biotech’s comeback company of the year.” Once thought to be a front runner in the race to the CAR-T finish line, Juno grabbed headlines following the death of five patients during the clinical trial of its lead program. But as recent data has shown, Juno is still in the race. Its latest CAR-T therapy targeting patients with large B-cell lymphoma has industry experts taking another look at the company. We’re excited to see what 2018 brings for Juno.
We were excited to see the news, reported at the 2017 American Society for Hematology (ASH) Annual Meeting, that patients treated with the newly approved CAR-T therapies, Kymriah and Yescarta, have demonstrated durable remissions. Gilead reported that after a year of follow-up, 40 percent of patients with diffuse large B-cell lymphoma continued to show complete responses to Yescarta. Meanwhile, Novartis reported that 6 months after treatment with Kymriah, also for diffuse large B-cell lymphoma, 30 percent had no detectable sign of cancer, and another 7 percent experienced a partial response. We’re staying posted for additional data to emerge in the months and years to come. (Speaking of the ASH annual meeting, don’t miss the blog entry that recaps our favorite moments from the conference!)
We would be remiss in overlooking what was far and away the biggest biotech news of December — the approval of the first gene therapy for an inherited disease. In late December, the FDA announced the approval Spark Therapeutic’s drug Luxturna, a one-time treatment for biallelic RPE65 mutation-associated retinal dystrophies, some of which can cause permanent blindness. This is a truly inspiring advance for medicine, and one that bodes well for the future of the entire biotech industry.