On August 23, 2022 Northwest Biotherapeutics (OTCQB: NWBO) ("NW Bio"), a biotechnology company developing DCVax personalized immune therapies for solid tumor cancers, reported that it has received approval from the UK Medicines and Healthcare Products Regulatory Agency (MHRA) for the Company’s Pediatric Investigation Plan (PIP) (Press release, Northwest Biotherapeutics, AUG 23, 2022, View Source [SID1234618570]). The development, regulatory review and regulatory approval of a PIP is a pre-requisite for application for approval of a new medicine for adult patients, such as DCVax-L.

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The Company’s approved PIP includes 2 clinical trials: one for newly diagnosed pediatric high grade glioma (HGG), and one for recurrent pediatric HGG. In each of the 2 pediatric trials, 24 patients will be treated with DCVax-L on the same treatment schedule as in the Company’s Phase III trial in adult glioblastoma patients.

The primary endpoint for each of the 2 pediatric trials will be overall survival, determined by comparing the survival of DCVax-L treated patients to matched contemporaneous external controls. The external controls will be identified using the same methodology as was used to pre-specify the external controls in the Statistical Analysis Plan for the Company’s Phase III trial in adult patients.

Under applicable UK law, when a new medicine is developed for adult patients, that medicine must also be tested for potential application to pediatric patients. The sponsor must develop an overall Plan to select the specific form or stage of the disease to be treated, to adapt the dosing and administration of the medicine for pediatric physiology, and to evaluate the safety and efficacy of the medicine in pediatric patients. Further, the Plan must include not just general focus areas, aims and approaches — it must include the full design of the specific clinical trials to be carried out, including all aspects required for clinical trial approvals, such as the patient population, eligibility criteria, stage of disease, treatment regimen, trial design and endpoints.

The Plan developed by the sponsor must go through a series of stages of regulatory review and comment to reach a final approval by regulators. This process can typically take more than a year.

The final regulatory approval of the PIP must be obtained before a sponsor may submit a Marketing Authorization Application (MAA) for approval to commercialize the new medicine for adult patients. The approval may include a deferral allowing the pediatric clinical trials to actually be carried out after the MAA has been submitted, but the PIP approval itself must have been received before an MAA can be filed and go through compliance check.

Northwest Biotherapeutics worked with expert consultants for months to develop a PIP tailored for application of DCVax-L to pediatric cases of HGG. The Company submitted its proposed PIP to the MHRA in February 2022, and has been going through the regulatory review process since then. On August 17, the Company received final approval of the PIP from the MHRA.

The Company’s approved PIP includes a deferral under which the pediatric trials are anticipated to be undertaken after an MAA application has been submitted.