Expanded Access Policy

Clinical Trials and Early Access

Clinical trials, and the subsequent approval of a therapy, can provide access to new therapies for patients in need. Patients interested in an investigational product that has not yet been approved are encouraged to speak with their physician to be evaluated for possible participation in a Savara clinical trial. Early access to investigational products or therapies can provide some patients who have serious or life-threatening diseases or conditions with access to investigational products that are not yet approved by local regulatory agencies outside of a clinical trial.

Savara has launched the global Savara Early Access Program and has partnered with WEP Clinical (a specialist early access service provider). An early access program might be called an expanded access program, particularly in the United States. Other terms for early or expanded access include compassionate use, named-patient use, or single-patient access.

Where to Find Information About Savara’s Early (aka Expanded) Access Program

You can find information about Savara’s clinical trials, including the status of patient recruitment, by searching for “Savara” at www.clinicaltrials.gov. You can find information about Savara’s early access program for molgramostim for eligible patients with autoimmune pulmonary alveolar proteinosis (aPAP) at www.clinicaltrials.gov.

Early Access Review Criteria and Process

When reviewing requests for access to molgramostim outside of a clinical trial, Savara considers guidance from the U.S. Food and Drug Administration (FDA) and other regulatory authorities as well as the following information:

1. The patient’s diagnosis, presentation of clinical symptoms, and access to satisfactory alternative treatment options (e.g., the patient must have a confirmed diagnosis autoimmune pulmonary alveolar proteinosis based on a positive GM-CSF autoantibody test with clinical symptoms and no access to satisfactory alternative treatment options)
2. The patient’s previous and current enrollment status in a clinical trial, including Savara’s IMPALA-2 clinical trial
3. The potential benefits of treatment with molgramostim versus the potential risks, as determined by Savara’s medical team
   • The medical team may request additional information from the patient’s treating physician in order to make this determination
4. The availability of supply of molgramostim to support early access as well as Savara’s ongoing clinical trials and development programs
5. Local laws and regulations about expanded access

Savara will review requests on a case-by-case basis according to the above information, as well as the inclusion and exclusion criteria in the expanded access program protocol (if applicable). Requests for access are not a guarantee that access to molgramostim will be granted.

Early Access Contact Information

Savara’s policy on expanded access will be reviewed on a regular basis and as more information and clinical trial data on the safety and efficacy of molgramostim become available. To request medical information about expanded access to molgramostim, physicians should contact SavaraEAP@WEPClinical.com, and a response will be received within 3 business days. All requests must be submitted by a licensed pulmonologist. Additional information about early access can be found on the Reagan-Udall Foundation Expanded Access Navigator website.