At Applied Therapeutics we recognize the difficulty of receiving a rare disease diagnosis for you or someone in your family. Our goal is to support patients and families as they explore what options may be available to help.

We demonstrate our commitment to patients and families by developing potential new therapies for the treatment of rare diseases and making them available to patients when and as allowed by regulatory authorities.  We work hard to get therapies through the approval process to make them generally available as soon as possible.  

As an important part of our commitment to patients and their families, Applied Therapeutics can make available some of its investigational therapies to qualified patients in the USA under certain circumstances of critical and compelling need when factors support this use through our Expanded Access program or in a clinical study.  Early access, sometimes called “compassionate use” or in the United States, Expanded Access, is a process that may help patients obtain investigational therapies that are not yet approved by the country’s regulatory agencies (e.g., the Food and Drug Administration (FDA)) outside of a clinical study.

Though Applied Therapeutics can make its investigational therapies available through Expanded Access, the primary preferred mechanism for Access to investigational therapies is by participation in a clinical study.  An investigational therapy is a potential new therapy that has not yet been approved by regulatory authorities such as the FDA in the United States or the European Medicines Agency (EMA) in Europe. Clinical studies collect the information necessary to understand the safety and efficacy of the potential therapy allowing for regulatory authority review and potential approval.  Ultimately, we believe that approval by regulatory authorities gives the opportunity for the broadest access because regulatory approval allows physicians to prescribe the therapy to any patients who might benefit.


To participate in a clinical study, you/your child must meet certain eligibility criteria.  You should discuss participation in a clinical study with your physician who knows your medical history, current status and can discuss if you may be able to join a clinical study.

For information on ongoing Applied Therapeutics clinical studies:

Visit and search registered clinical studies for the disease and/or the name of the Applied Therapeutics investigational product.

If you locate a clinical study with a participating site that you are interested in, please contact Applied Therapeutics via email at


At Applied Therapeutics, we recognize that some patients with serious or immediately life-threatening diseases may not be able or eligible to participate in clinical studies and may not have other treatment options for their condition.  Applied Therapeutics evaluates requests for individual patients to receive investigational therapies outside of a clinical study on a case-by-case basis. Requests for expanded access must be made by the patient’s physician.  For more information please visit:

As a company, Applied Therapeutics is committed to attempting to help and support patients where possible.  Unfortunately, not every situation will be one where we can help.  We greatly appreciate the challenges patients and families face and hope we can support them whenever possible.


Applied Therapeutics’ decisions for Expanded Access will be made on a fair and equitable basis for each investigational therapy. Each disease we are studying may have different specific criteria due to the differences in urgency and known data on safety and efficacy.   We consider the following when reviewing a request:

Serious manifestation of disease

  • Whether you/your child has a serious manifestation of a condition which is potentially irreversibly disabling or immediately life-threatening.

Not able to join a clinical study

  • Eligibility requirements or geographical limitations to participate in an ongoing clinical study.

No appropriate treatment options

  • Availability of other, appropriate treatment options. For example, if you/your child has tried standard treatments without success, exhausted all reasonable available treatment options, or can no longer tolerate existing treatment, you/your child may be an appropriate candidate.

Evidence of possible benefit

  • Clinical and/or scientific evidence of possible benefit from the investigational therapy for your/your child’s condition.

Benefit of treatment outweighs risks

  • Disease or symptoms that suggests the benefits of treatment outweighs any known or potential risks so that the potential to help the patient is plausible, based on available information.

Adequate supply of therapy

  • Supply levels of the investigational therapy available to meet the request.

Appropriate local regulatory pathway

  • Whether regulatory authorities where you/your child live allow early access to investigational therapies through a locally appropriate regulation.

Other possible considerations

  • Other criteria, fully reviewed with the requesting physician, based on the complexity and diversity of situations for each patient and disease situation.

Country specific laws and regulations will apply and may affect availability of the investigational therapy in that country or region. Applied Therapeutics may restrict availability in some countries.


For Patients/ Caregivers/ Patient Advocates

Requests for Expanded Access to investigational therapies must be made by a qualified physician.  You and your family should discuss your situation with your treating physician including current status, available treatment options and reason for considering treatment with an investigational therapy.  Your physician can make the request for Expanded Access on your behalf.  It is important that Expanded Access requests are made by medically trained personnel to avoid delays.

For more information on Expanded Access you may visit the United States Food and Drug Administration website.

For Physicians / Healthcare Professionals

All requests for Expanded Access for a patient must be submitted by the patient’s treating physician.

The patient’s treating physician must provide adequate information to demonstrate that the patient meets the outlined considerations for Expanded Access.

The patient’s treating physician must be appropriately licensed, fully qualified to administer the investigational therapy and have the expertise and facilities for monitoring, managing and reporting any adverse events during the course of treatment.  The patient’s treating physician must be willing to meet and follow all applicable legal and regulatory requirements for Expanded Access while caring for the patient receiving investigational therapy and must comply with all applicable reporting requirements.

Qualified physicians can submit a request by contacting Applied Therapeutics at

For more information on Expanded Access for Patients and Physicians in the United States, you may visit the Food and Drug Administration (FDA) website.


Applied Therapeutics will review the request and supporting documents from a treating physician to determine the urgency and need of the request. We are committed to acknowledging receipt of a request within 5 business days. We aim to respond to the request including our rationale as promptly as possible so that you and your family can explore all possibilities.

If the disease or condition is one we have not treated or are not studying, our ability to help may be limited. In general, prior treatment of the disease with the investigational therapy is needed for Applied Therapeutics to consider Expanded Access.

As a company, Applied Therapeutics is committed to attempting to help and support where possible.