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In the European Union, medicinal products are authorized following a similar demanding process as that required in the United States
and applications are based on the ICH Common Technical Document. Prior to submitting a European Marketing Authorization Application, or MAA, it is necessary to gain approval of a detailed Pediatric
Investigation Plan, or PIP, with the European Medicines Agency's Pediatric Committee, or PDCO. After gaining PIP approval, medicines can be authorized in the European Union by using either the
centralized authorization procedure or national authorization procedures.
Under the centralized procedure, after the EMA issues an opinion, the European Commission issues a single marketing authorization valid
across the European Union, as well as Iceland, Liechtenstein and Norway. The centralized procedure is compulsory for human medicines that are: derived from biotechnology processes, such as genetic
engineering; contain a new active substance indicated for the treatment of certain diseases, such as HIV/AIDS, cancer, diabetes, neurodegenerative disorders or autoimmune diseases and other immune
dysfunctions; and officially designated orphan medicines. For medicines that do not fall within these categories, an applicant has the option of submitting an application for a centralized marketing
authorization to the EMA, as long as the medicine concerned is a significant therapeutic, scientific or technical innovation, or if its authorization would be in the interest of public health.
There are also two other possible routes to authorize medicinal products in several countries, which are available for products that
fall outside the scope of the centralized procedure:
- Decentralized procedure. Using the decentralized
procedure, an applicant may apply for simultaneous authorization in more than one European Union country of a medicinal product that has not yet been authorized in any European Union country and that
does not fall within the mandatory scope of the centralized procedure.
- Mutual recognition procedure. In the mutual recognition
procedure, a medicine is first authorized in one European Union Member State, in accordance with the national procedures of that country. Thereafter, further marketing authorizations can be sought
from other European Union countries in a procedure whereby the countries concerned agree to recognize the validity of the original, national marketing authorization.
we believe that our development program, proposed Phase 3 trial design, and overall non-clinical and clinical data package could support future regulatory approval of
PRT-201 in the European Union, we have not submitted such information to the European Union for their review.
Like the FDA, the EMA, the competent authorities of the European Union Member States and other regulatory agencies regulate and inspect
equipment, facilities and processes used in the manufacturing of pharmaceutical and biologic products prior to approving a product. If, after receiving clearance from regulatory agencies, a company
makes a material change in manufacturing equipment, location, or process, additional regulatory review and approval may be required. Once we or our partners commercialize products, we will be required
to comply with cGMP, and product-specific regulations enforced by, the European Commission, the EMA and the competent authorities of European Union Member States following product approval. Also like
the FDA, the EMA, the competent authorities of the European Union Member States and other regulatory agencies also conduct regular, periodic visits to re-inspect equipment, facilities, and processes
following the initial approval of a product. If, as a result of these inspections, it is determined that our or our partners' equipment, facilities, or processes do not comply