Unique trends are occurring in the pharmaceutical pipeline that would have been unheard of just five years ago. One agent coming on the market may cost up to $1 million for a one-time infusion, and a new agent derived from cannabis may be the first of many such products for a host of illnesses.
Perry Cohen, Managed Healthcare Executive editorial advisor, and CEO of the Pharmacy Group consulting firm, says some remarkable agents will be coming on the market that will significantly help individuals who previously had no treatment options.
Here, industry experts share more information about noteworthy new drugs in the pipeline.
- What it is: AveXis, Inc., which is a clinical-stage gene therapy company, says it is using an adeno‐associated virus vector (AAV9) containing the SMN1 transgene for the treatment of spinal muscular atrophy (SMA) infusion (one-time). The company says about 7,200 infants have SMA Type 1, which represents infants from birth to six months of age. Babies with SMA Type 1 have difficulty breathing and swallowing. They cannot sit, crawl or walk and only 8% of babies with SMA Type 1 survive to 20 months of age.
- Potential impact: All patients in the therapeutic dose cohort in the phase 1 trial were alive and event-free at 24-month follow-up. Patients observed in long-term follow-up from the phase 1 trial continued to gain strength and achieve new milestones, including two additional patients who could stand with assistance.
- Status: Orphan drug. Biologics License Application (BLA) filing possible in mid-2018 with approval in late-2018 or early-2019, according to Christopher Peterson, PharmD, director, clinical evaluation and policy, Express Scripts.
- Cost estimate: $750,000 to $1 million for the one-time infusion, according to Peterson.
AVXS-101 could be the first one-time gene replacement therapy for SMA, a disease which results in early death or lifelong disability with considerable healthcare costs. It is expected to be available in the U.S. in 2019.
Sukumar Nagendran, is chief medical officer and senior vice president of AveXis, which is developing this product. He expects to submit a request for a pre-BLA meeting with FDA by the end of June to discuss the regulatory path for AVXS-101. “We will be able to provide more guidance after the meeting minutes are received, typically approximately a month after the meeting,” says Nagendran