Physician Views: Epidiolex breaks new regulatory ground as first cannabidiol-derived seizure therapy

On Monday, the FDA approved GW Pharmaceuticals' Epidiolex (cannabidiol) oral solution for the adjunctive treatment of seizures linked to Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS) in patients at least two years old. CEO Justin Gover remarked that the approval marks "a historic milestone, offering patients and their families the first and only FDA-approved [cannabidiol] medicine to treat two severe, childhood-onset epilepsies."

Analysts have modelled peak blockbuster sales for Epidiolex, though such an outcome will be dependent on its broader use in refractory epilepsy patients not diagnosed with LGS or DS. The language included in Epidiolex's label provides room for optimism on this front, they add, so we are snap-polling US-based neurologists to get a better view on how they intend to use this therapy…

The FDA has approved Epidiolex (cannabidiol) oral solution for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS) in patients two years of age or older. Epidiolex is the first prescription pharmaceutical formulation of highly-purified, plant-derived cannabidiol (CBD), a cannabinoid lacking the high associated with marijuana, and the first in a new category of anti-epileptic drugs (AEDs).

How aware are you of Epidiolex and its clinical data?

Not aware

Slightly aware

Moderately aware

Very aware

Extremely aware

The effectiveness of Epidiolex for the treatment of seizures associated with LGS was established in two randomised, double-blind, placebo-controlled trials in patients aged two to 55 years.

In the first study, monthly seizure frequency declined by 44% in patients given Epidiolex, compared to 22% in those who received placebo. In the second study, low (10mg/kg/day) and high (20mg/kg/day) doses of Epidiolex were associated with 37% and 42% reductions in seizure frequency, respectively, versus a decline of 17% in the placebo arm.

In a Phase III study for DS, Epidiolex was associated with a 39% median reduction in monthly convulsive seizures, versus 13% for placebo.

In each study, the difference in seizure frequency became apparent in the first month of treatment and persisted throughout the 14 week treatment period.

Based on these data, how do you expect Epidiolex to be adopted as a treatment for LGS and DS patients?

No adoption

Marginal adoption

Moderate adoption

Significant adoption

Very significant adoption

According to its label, "Epidiolex is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS) in patients 2 years of age and older." There is speculation this language is consistent with broader use of Epidiolex, which will permit reimbursement in a wider variety of refractory seizure disorders.

How do you expect Epidiolex to be adopted as a treatment for refractory epilepsy patients not diagnosed with LGS or DS?

No adoption

Marginal adoption

Moderate adoption

Significant adoption

Very significant adoption

And in terms of total Epidiolex use, approximately what proportion do you estimate will come from refractory epilepsy patients not diagnosed with LGS or DS?

Less than 10 percent

10-25 percent

25-50 percent

50-75 percent

More than 75 percent

Will Epidiolex's status as a cannabinoid 'meaningfully' limit uptake during its first few years on the market?

No

Yes - it will meaningfully limit uptake across all indications

Yes - it will meaningfully limit uptake in non-LGS and DS patients   

Results and related analysis will shortly be published for FirstWord Pharma PLUS subscribers to read, with the opportunity for non-FirstWord Pharma PLUS subscribers to purchase these findings. To be notified when poll results and analysis become available, please click here.

As always, FirstWord would very much like to receive your feedback and suggestions. Note: FirstWord Polls are powered by Medefield MedePolls, a fast-turnaround service to conduct instant polls of up to five questions with guaranteed samples that include physicians from dozens of specialties and over 100 markets. To conduct this poll with a different audience, or an entirely different poll, contact us at info@firstwordpharma.com.

To read more Physician Views articles, click here.