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epidiolex success rate

In total, three pivotal, multinational, double-blind, placebo-controlled, Phase 3 trials, all part of the GWPCARE series, were included in the review. All these trials were built on the foundation laid by a previous prospective, multicenter, open-label Expanded Access Program (EAP) trial designed to evaluate the effects of Epidiolex in a group of 162 patients with treatment-resistant epilepsy.

“These trials revealed CBD’s efficacy in reducing the frequency of the primary seizure type over a 14-week period compared with placebo, providing compelling evidence for the role of pure, plant-derived CBD in the management of DS and LGS,” the researchers said.

It is carbohydrate neutral and compatible with ketogenic diets — a diet high in fats and low in carbs that has been shown to reduce the frequency of seizures and improve cognitive function in children with Dravet syndrome.

Researchers in this study reviewed the main findings of these trials regarding the safety, efficacy, and tolerability of Epidiolex for the treatment of children with Dravet and Lennox-Gastaut syndromes.

Further chemical analysis also revealed that when used together with another anti-epileptic medication, such as Onfi (clobazam) or Depacon (valproate sodium), Epidiolex may increase the risk of side effects and should, therefore, be administered with caution.

Previous clinical trials have found that Epidiolex effectively reduces the frequency of epileptic seizures when used in combination with other anti-epileptic medications.

“The frequency and severity of seizures have profound impacts on quality of life, risk for injury (eg, convulsive seizures in [Dravet syndrome], drop seizures in [Lennox-Gastaut syndrome]), health care use, and increased risk for mortality,” the researchers wrote.

In addition, patients are given Epidiolex on a gradually increasing dosage level, starting from 2.5 mg/kg taken twice daily and then staying on 5 mg/kg twice daily for some time before titrating up to as high as 20 mg/kg. Gangolli said the observation period will likely last several months for existing patients, and the likelihood of their reaching maximum dosages is “relatively remote.”

First, pent-up demand. Prescriptions from previously diagnosed patients waiting for the launch delivered a surge of scripts carrying into Q1, Gangolli said. Plus, many commercial and state Medicaid programs only implemented coverage decisions in January, pushing scripts into the new year, he said.

The company now plans to file for that new approval in the fourth quarter, aiming for a potential launch in 2020. It estimates TSC affects 40,000 to 80,000 people in the U.S. and about 1 million to 2 million worldwide. That means a TSC approval would roughly double Epidiolex’s current market, J.P. Morgan analyst Cory Kasimov figures.

Given the “huge influx of patients,” Gangolli cautioned about looking at quarter-on-quarter growth when Q2 results come around. Some unusual factors played into the Q1 beat that neither execs nor analysts seem to have expected.

In its first full quarter on the market, Epidiolex racked up sales of $33.5 million, more than double analyst expectations of $16 million. And positive results from a phase 3 trial have put it on track to an important market expansion.

The company is also seeing a higher percentage of adult patients than it had expected, which Gangolli attributed to payer deals that feature less restrictive prior authorization requirements, or no prior authorization at all. Because Epidiolex’s dosage level is weight-based, more adult patients could also translate into bigger sales.

“Our expectation was and continues to be that physicians will prescribe to gain experience with an initial set of patients and observe those patients over a four- to six-month period as to therapeutic effect, before determining how to utilize Epidiolex in a broader set of patients,” Gangolli said, adding that it has now entered that evaluation period.

Data were presented on all 261 patients who had at least 12 weeks treatment. Treatment was open label. Of these 261 patients, 135 patients had also reached 36 weeks treatment at the time of data analysis. Information collected on all seizures (convulsive and non-convulsive) is reported for each patient. Data are presented showing the median change in seizure frequency compared to a 4-week baseline period.

Safety Data

Safety data were made available on 313 patients (261 patients with 12 weeks treatment effect data plus 52 additional patients for whom 12 week treatment effect data are not yet available or who withdrew from treatment) and represent approximately 180 patient-years of exposure to Epidiolex.

GW Epidiolex Development Program – Dravet syndrome, LGS and TSC

GW is currently conducting formal development programs in the treatment of three indications; Dravet syndrome, LGS and TSC. GW has to date received Orphan Drug Designation and Fast Track Designation from the FDA for Epidiolex for the treatment of Dravet syndrome, as well as Orphan Designation from the European Medicines Agency (EMA). GW has also received Orphan Drug Designation for Epidiolex for the treatment of LGS. GW is conducting two Phase 3 trials in Dravet syndrome and two Phase 3 trials in LGS. GW expects to report initial top-line results from these trials in 2016. GW also expects to commence Phase 3 clinical development of Epidiolex in TSC in early 2016.