Nouryant garners negative CHMP opinion for Parkinson's disease

Regulatory NewsRegulatory News | 23 July 2021 |  By 

The human medicines committee of the European Medicines Agency (EMA) recommended two new medicines for approval during its July meeting; the committee also recommended four extensions of therapeutic indication and issued a negative opinion on one medicine.
Genzyme’s Nexviadyme (avalglucosidase alfa) was recommended by the Committee for Medicinal Products for Human Use (CHMP) to treat glycogen storage disease type II, also known as Pompe disease. Nexviadyme had received orphan designation, securing 10 years of market exclusivity for the therapy in the EU.
Imatinib, a hybrid medicine sponsored by KOANAA Healthcare, received a positive recommendation to treat both leukemia and gastrointestinal stromal tumors. “Hybrid applications rely in part on the results of pre-clinical tests and clinical trials of an already authorised reference product and in part on new data,” explained CHMP in making the announcement.
Nouryant (istradefylline), a proposed treatment for adults with Parkinson’s disease sponsored by Kyowa Kirin Holdings, garnered a negative opinion from CHMP, the committee’s second negative opinion of 2021 for a new medicine.
An adenosine A2A receptor antagonist, Nouryant was meant to be an orally taken add-on to levodopa for patients experiencing “off” periods. CHMP noted that its negative opinion was based on the fact that only half of the eight studies presented by the sponsor actually showed a reduction in “off” time with Nouryant, with no dose-response effect seen. Also, the two studies that involved EU patients were among those with negative results.
Otsuka’s Deltyba (delamanid) received a positive recommendation for use as part of a regimen to treat multi-drug resistant tuberculosis in infants with a body weight as low as 10 kg, extending the indication to this age and weight group. Ultomiris (ravulizumab), Alexion Europe’s treatment for paroxysmal nocturnal hemoglobinuria, received a similar recommendation for extension of indication to pediatric patients weighing at least 10 kg.
GlaxoSmithKline’s Volibris (ambrisentan) received a CHMP recommendation to extend the drug’s use to treat pulmonary arterial hypertension to the pediatric population aged at least 8 years.
Finally, Gilead’s Vosevi (sofosbuvir / velpatasvir / voxilaprevir), a medicine to treat chronic hepatitis C virus infection, was recommended to treat patients weighing at least 30 kg and aged 12 years and up; it had previously only been authorized to treat adults.
Other CHMP actions

In July, CHMP also affirmed that it found no link between acute myeloid leukemia (AML) and the viral vector in Zynteglo (bluebird bio, betibeglogene autotemcel). That medicine shares a viral vector with an investigational product that was associated with two cases of AML.
CHMP has concluded its review of this issue for Zynteglo: “After examining all the evidence, it was clear that more plausible explanations for the AML cases included the conditioning treatment the patients received to clear out bone marrow cells and the higher risk of blood cancer in people with sickle cell disease,” the indication for the investigational therapy, wrote the committee.
CHMP declined to extend the indication for Addmedica’s Siklos (hydroxycarbamide) to include treatment of severe chronic anemia in patients with sickle cell syndrome, citing missing data in the sponsor’s submission.
Roche withdrew is marketing authorization application for Tecentriq (atezolizumab) to treat early or locally advanced triple-negative breast cancer (TNBC) after EMA’s evaluation of data presented from the sponsors. Roche responded to questions from EMA, but the agency judged that “there were still some unresolved issues” after taking those responses into consideration.
In its withdrawal letter for Tecentriq for the TNBC application, Roche stated that it was doing so because CHMP considered that the study data provided “do not allow the committee to conclude on a positive benefit-risk balance for the use of Tecentriq in the proposed indication stated above.”
Flynpovi, Cancer Prevention Pharma’s formulation of eflornithine and sulindac to treat familial adenomatous polyposis, was handed a negative opinion by CHMP in June. At that time, the committee cited the lack of a control arm in studies and the fact that there is no evidence that either drug alone is effective in treating the condition, as well as insufficient long-term safety data. The firm has requested a re-examination of that decision. “Upon receipt of the grounds of the request, the Agency will re-examine its opinion and issue a final recommendation,” wrote the committee in its Thursday update.
CHMP July highlights


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