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Kinesys supports you obtaining orphan designation and beyond

Special products need special attention from people who understand. Orphan medicinal products (OMPs) almost always falls into this category. OMPs can be life-saving treatments but their development is often fraught with difficulty. Taking an OMP to market requires specific knowledge and expertise.

Kinesys has worked extensively on the development and registration of a range of OMPs across a broad range of rare diseases.

At Kinesys we appreciate the development, regulatory and commercial challenges associated with developing OMPs and we advise on expedited development pathways for these special products.

We fully understand what constitutes a robust Orphan Designation Application and have significant expertise in constructing well written and concise arguments to support key areas such as medical plausibility and significant benefit.

We intimately understand the requirements for Significant Benefit – often a prerequisite for Orphan Designation specific to the EU – and for maintaining Orphan Designation at the time of the MAA, an increasing challenge under the evolving legislation.

Our team of experts is continuously appraised of current and possible future legislative changes around the designation and approval of OMPs, and the implications of these changes. Kinesys has good relations with key members of the Committee for Orphan Medicinal Products (COMP).

Experience of presenting complex arguments at Oral Explanations to the COMP, resulting in successful outcomes for our clients.

Kinesys also has a successful track record with Orphan Drugs at US FDA.

Contact us to hear how we can care for your rare disease product.

Orphan Medicinal Products – Key Benefits

  • Provides incentives to companies to foster the development of products in diseases where the market has traditionally been considered commercially unattractive.
  • Offers hope of improved quality of life to approximately 30 million patients in the EU and 30 million in the US who suffer from debilitating rare diseases.
  • Orphan designated products are eligible for significant fee reductions for Scientific Advice (protocol assistance) and Marketing Authorisation Applications, as well as other pre- & post-authorisation activities. In USA, Orphan products are eligible to special financial incentives, funding for clinical investigations, market exclusivity (7 years), tax credit and a waiver of Prescription Drug User Fees.
  • All Marketing Authorisation Applications for designated OMPs are assessed through the centralised procedure with access to all EU member state markets and the potential to apply for conditional approval.
  • Opens up grant funding opportunities from the European Commission and other national sources.This is also true in USA, and notably clinical trials may attract funding.
  • Authorised OMPs benefit from 10 years market exclusivity in EU and 7 years in USA. In EU this is extended by a further 2 years for products complying with an agreed Paediatric Investigation Plan at time of designation.

Samples of projects Successfully Completed

A BTK inhibitor for LPL, CLL and MCL

A hormonal product for high risk pregnancies

A NCE for prevention of oral mucositis

An I.V. prostaglandin E1 for acute respiratory distress syndrome

A biological agent for pancreatic cancer

A Microbiota Transplant Product Recurrent Clostridium Difficile Infection

An opioid agent for uremic pruritus

A respiratory product for premature infants, two indications:

acute respiratory distress syndrome

meconium aspiration syndrome

An anti-epileptic drug for Lennox-Gastaut syndrome

Two stem cell agents, both for a GI condition

A re-profiled product for prevention of GVHD

A bioterrorism vaccine for ricin poisoning

Various oncology and haematology agents for conditions such as:

chronic lymphocytic leukemia

cutaneous T cell lymphoma

acute myeloid leukemia

brain metastases secondary to breast cancer

glioma

ovarian cancer

“A Disease May Be Rare, Hope Should Never Be”

Hunter Pageau, 12-year-old boy suffering from a motor neuron disease that often kills affected children before their first birthdays.