Dedicated to developing safe and effective medicines to improve the lives of patients and their families

Expanded access to Pharnext investigational drugs (for US only)

Pharnext develops innovative new medicines designed to address unmet medical needs of patients in major therapeutic areas. We are focused on creating new medicines that address important, real-world concerns of patients and their families. We currently have investigational treatments in clinical trials for a number of different diseases, as described in the pipeline section.

Pharnext’s goal is to efficiently bring safe and effective medicines to all appropriate patients by conducting rigorous clinical trials and obtaining marketing approval by regulatory authorities, including the U.S. Food and Drug Administration (FDA). Prior to such approval, participation in one of our clinical trials is the optimal way for patients to gain access to our investigational medicines. However, we understand that some patients may not be able to participate in a clinical trial and may seek access to investigational treatments before they are approved. These situations are often referred to in various ways, including expanded access, early access, pre-approval access, compassionate use and emergency use.

If early data from clinical trials suggest that an investigational treatment can offer benefits for patients facing serious or life-threatening conditions, Pharnext may consider requests to provide access outside of a clinical trial. Our top priority when evaluating requests for expanded access to investigational medicines is to ensure that we are not putting patients at risk of unnecessary harm.

Pharnext will consider an expanded access program, or a single request for expanded access of an investigational medicine, only if all of the following criteria are met:

  • The patient’s licensed physician submits an unsolicited request for expanded access

  • The patient to be treated has a serious or immediately life-threatening illness and there is no satisfactory alternative therapy

  • There are no comparable or satisfactory alternative therapies approved and available to treat the disease or condition

  • There is preliminary clinical evidence of effectiveness and acceptable safety of the investigational treatment for that patient’s condition

  • The potential benefit of the investigational medicine to the patients is greater than the potential risk

  • Staff and other required resources are available to provide the product and there is an adequate supply of the investigational medicine

  • Providing the investigational medicine will not interfere with the timely completion of clinical trials that could lead to marketing approval and thereby could delay its availability to other patients

  • Expanded access will only occur in a country where Pharnext expects to file for regulatory approval of the product

Patients interested in obtaining access to a Pharnext investigational therapy must do so via their physician. Physicians should contact Pharnext Medical Information Center at All requests will be carefully considered, and a written or oral response will be provided to the treating physician within two weeks of receiving a request. For further information on available expanded access programs, visit and search “expanded access programs”.

Charcot-Marie-Tooth Patients
CMT&Me Study

If you’ve been diagnosed with CMT, you might like to consider participating in the CMT&Me international study. The app-based study is sponsored by Pharnext and has been developed in collaboration with Vitaccess, a digital health company, international CMT experts, and patient advocacy groups.

CMT&Me investigates the impact of all subtypes of the disease on patient lifestyle: The global burden of the disease including daily work and social lives, its natural history, current treatment options, and medicosocial and pharmacoeconomic effects.

Answers to your questions

Charcot-Marie-Tooth (CMT) diseases − named after the three doctors who first described them in 1886 − are genetic diseases that affect the peripheral nerves, which are the nerves that connect the central nervous system (brain and spinal cord) to the rest of the body by providing motor and sensory information. CMT diseases are relatively rare and affect about 1 in 2,500 people.

This guide has been designed to address the need for information for affected people and their families, taking into account the most common questions they have. It does not replace the explanations given by your neurologist, but can help you better understand and answer your initial questions.

Answers to your questions

Several studies carried out in recent years have demonstrated the very common existence of pain in Charcot-Marie-Tooth (CMT) disease. This pain is currently the subject of studies in order to better understand its mechanism and onset, its impact on the everyday life of patients and the measures that can be taken to relieve it [2-4, 6-8]. At the same time, healthcare teams are paying particularly close attention to it.

This seconde guide has been designed to answer the main questions that you or those around you have on the matter. It does not replace the explanations given by the healthcare professionals monitoring you but it may enable you to better prepare for your consultations with them and to better understand their explanations and any management proposed.

Patients groups
Our Treatment in Development

Pharnext is actively developing PXT3003 for the treatment of patients with Charcot-Marie-Tooth Type 1A disease.

Explore moreExplore
Next step