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Unique trends are occurring in the pharmaceutical pipeline that would have been unheard of just five years ago.
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.
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
Epidiolex (GW Pharmaceuticals)
Cannabidiol is a pharmaceutical formulation of purified cannabidiol. GW Pharmaceuticals reported in phase 3 studies that cannabidiol added to other antiepileptic therapies significantly reduced the frequency of seizures in patients with LGS and Dravet syndrome. If approved, this agent would be the first pharmaceutical formulation of purified, plant-based CBD, according to GW Pharmaceuticals This product is a cannabinoid lacking the high associated with marijuana and it would be the first in a new category of anti-epileptic drugs, according to GW Pharmaceuticals. Aimee Tharaldson, PharmD, senior clinical pharmacist advisor, Express Scripts says cannabidiol looks like a promising drug that could be a gamechanger for these patients. “There is a need for new treatment options for these patients. In three pivotal clinical studies, Epidiolex demonstrated strong efficacy in reducing seizure frequency in patients with LGS and DS,” Tharaldson says.
Larotrectinib (LOXO-101) (Loxo Oncology)
Most cancer drugs are targeted to specific organs or locations in the body. Larotrectinib is the first cancer drug to receive FDA breakthrough therapy designation for patients with a specific fusion of two genes in the cancer cell, no matter what cancer type, according to Laetsch. While the TRK fusions occur in only a small percentage of common adult cancers, they occur frequently in some rare pediatric cancers, such as infantile fibrosarcoma, cellular congenital mesoblastic nephroma, and papillary thyroid cancer, says Laetsch.
“I do think this is a gamechanger,” Laetsch says. “Larotrectinib has received breakthrough designation for the treatment of patients with a TRK fusion positive solid tumor, regardless of the type of tumor in which that fusion occurs. I see this as the next big step in the development of precision medicine and molecularly targeted therapy.”
Aimovig (erenumab – Amgen/Novartis)
“The new CGRP inhibitor class of medications will provide a treatment option for patients with chronic or episodic migraines,” says Christopher Peterson, PharmD, director, clinical evaluation and policy, Express Scripts. “Many of these patients are resistant or intolerant to currently available therapies. As a debilitating condition, decreasing the number of migraine days in patients with chronic or episodic migraines is an important advancement for these patients.”
“I would categorize this therapy as an ‘incremental improvement’ as there are many currently available therapy options for treating diabetes, including the SGLT-2 inhibitors that have a somewhat similar mechanism of action, says Peterson. However, diabetes is a large therapy class (the number two therapy class, according to Express Script’s 2017 “Drug Trend Report”) affecting about 30 million Americans. “As a result, the introduction of effective therapies within the class have the potential to achieve blockbuster status.”
Joshua Cohen, MD, global therapeutic area lead for migraine and headache at Teva, says his team is also studying fremanezumab for the prevention of chronic and episodic cluster headache as part of the phase III ENFORCE clinical research program. “Additionally, Teva has recently initiated a fremanezumab phase 2 clinical program for the treatment of post-traumatic headache disorder,” says Cohen.
Jed Black, MD, senior vice president of sleep medicine at Jazz Pharmaceuticals and an adjunct professor, Stanford University Medical Center, Stanford Center for Sleep Sciences and Medicine, says Jazz expects to launch solriamfetol in the U.S. in the first quarter of 2019.
“We believe solriamfetol will provide a meaningful option for patients living with excessive sleepiness due to narcolepsy or OSA once approved,” says Black.
John Schieszer, MA, is an award-winning national journalist and radio and podcast broadcaster of “The Medical Minute.” He also produces the Managed Healthcare Executive podcast, “News You Should Know.”