We'll assume you're ok with this, but you can opt-out if you wish. GenSight Biologics’ lead product candidate, LUMEVOQLeber Hereditary Optic Neuropathy (LHON) is a rare maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells that results in brutal and irreversible vision loss that can lead to legal blindness, and mainly affects adolescents and young adults. Powered by Madgex Job Board Software These questions will be the basis for a planned meeting between EMA and Ombudsman case-handlers in September. O’Reilly has begun the process of gathering evidence after writing to the Executive Director, Guido Rasi, at the EMA. The European Medicines Agency’s (EMA) is under investigation by European Ombudsman, Emily O’Reilly after she raised concerns that EMA’s pre-submission meetings with sponsors could influence the agency’s decisions about marketing authorisations (MAs). The letter also raises questions on the rules governing contact between Rasi’s staff and manufacturers, and whether it allows the marketing authorization decision makers at EMA to participate in pre-submission meetings and whether there are sufficient checks in place to ensure decisions are objective.To access the full letter from the European Ombudsman, please click This website uses cookies to improve your experience. LHON is associated with painless, sudden loss of central vision in the 1RESCUE and REVERSE are two separate randomized, double-masked, sham-controlled Phase III trials designed to evaluate the efficacy of a single intravitreal injection of GS010 (rAAV2/2-The primary endpoint will measure the difference in efficacy of GS010 in treated eyes compared to sham-treated eyes based on Best‑Corrected Visual Acuity (BCVA), as measured with the ETDRS at 48 weeks post-injection. document at a pre-submission meeting. The EMA pre-submission meeting is a procedural milestone in the preparation of a marketing authorization application (MAA). All rights reserved. In the centralized procedure, the process is determined by the EMA.

Additionally, a categorical evaluation with a responder analysis will be evaluated, including the proportion of patients who gain 15 ETDRS letters from baseline and nadir and the proportion of patients with Snellen acuity of >20/200. In October 2018, the Ombudsman launched a consultation on EMA’s engagement with medicine developers in pre-submission activities. Scientific Advice & Pre-Submission Meetings. The program and initial consultation with FDA is free and is designed to support successful registration of your medical device. Complementary vision metrics will include automated visual fields, optical coherence tomography, and color and contrast sensitivity, in addition to quality of life scales, bio‑dissemination and the time course of immune response.

O’Reilly has begun the process of gathering evidence after writing to the Executive Director, Guido Rasi, at the EMA.

The EMA emphasises the importance of p re-submission meetings between applicants and the EMA/(Co-) Rapporteur. A topic should only be proposed for discussion, when applicant’s questions are not fully answered by the pre-submission or other available guidance documents, due to certain

The FDA Pre-Submission program is free and often overlooked, but can save sponsors valuable time and money. As you prepare your submission, a Sponsor can seek scientific in one of two ways: (1) at a national level from their individual member states regulatory authorities, or (2) from the EMA’s CHMP. Both trials have been adequately powered to evaluate a clinically relevant difference of at least 15 ETDRS letters between treated and untreated eyes adjusted to baseline.The secondary endpoints will involve the application of the primary analysis to best‑seeing eyes that received GS010 compared to those receiving sham, and to worse‑seeing eyes that received GS010 compared to those that received sham. The secondary objective is to describe the relationship between genetic, lifestyle and/or environmental factors and the expression of the LHON phenotype.The first subject was enrolled on 3 January 2018, and enrollment is targeted to be completed in early Q2 2020.© 1985 - 2020 BioSpace.com. The EMA pre-submission meeting is a procedural milestone in the preparation of a marketing authorization application (MAA). The organization of a pre-submission meeting can also be considered within the Perhaps this information was designed to provide assurance to the companies investors, but it bothered O’Reilly; her concerns are that the public may view this as bias so she is asking EMA to take proactive measures to reduce such perceptions.In the letter to Rasi, O’Reilly’s questions have a strong focus on transparency and include the possibility of EMA publishing detailed minutes of pre-submission meetings.