Minoryx is a clinical stage biotech company committed to the development of safe and effective novel therapies for severe and life-threatening orphan CNS diseases with high unmet medical needs. We are a science-driven company committed to putting patients first. Our goal is to provide access to our investigational therapies at the appropriate time and in the correct manner for patients.
Clinical trials and the subsequent timely generation of safety and efficacy data are the most effective way of ensuring review and decision making by Regulatory Authorities. This ultimately has resulted in access to new, safe and effective approved medicines for patients.
Expanded access refers to the use of an investigational therapy outside a clinical trial when the primary purpose is to diagnose, prevent, or treat a serious condition in a patient. We understand the need for expanded access programs, and we recognize the importance of having an appropriate policy.
FDA Orphan indication was granted for the company’s lead program, leriglitazone (MIN-102), a novel, selective peroxisome proliferator-activated receptor gamma (PPARγ) agonist, for the treatment of X-ALD. On 4 April 2018, Minoryx opened the IND for leriglitazone (company code MIN-102) with a double-blind, randomized, placebo-controlled study to assess the effect of leriglitazone on the clinical progression in male patients with Adrenomyeloneuropathy (AMN). Recently, the FDA granted Fast Track Status to leriglitazone for development in patients with X-ALD.
An Early Access program for leriglitazone may be opened if Minoryx determines that all the following criteria are met:
• The investigational drug is the subject of an active clinical development program and is not approved in any indication in the country concerned
• An adequate supply of the investigational drug exists to perform necessary clinical studies as well as to provide Early Access to patients who do not have alternative treatment options
• Early Access does not impede or compromise the clinical development or regulatory approval of the drug under investigation
• There are sufficient clinical data available with respect to both the investigational drug and the disease condition for which the application is being sought, to anticipate that any potential benefits from treatment are likely to outweigh any potential risks to the patient
At this time, Minoryx believes that participation in one of our clinical trials is the most appropriate way to access our investigational therapies. Minoryx continues to assess the eligibility requirements and criteria for Early Access to the investigational drug leriglitazone and will re-evaluate this policy from time to time.
If you have additional questions, please speak with your physician or contact clinical@minoryx.com. We anticipate acknowledging receipt of requests sent to this email within five business days.