Expanded Access Policy

Clinical Trials and Expanded 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. Expanded 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 approved by local regulatory agencies outside of a clinical trial.

Savara has launched the global Savara Early Access Program and has partnered with Uniphar (a specialist expanded 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 Clinical Trials and Early (or Expanded) Access Program

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

Expanded 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 general criteria:

1. The patient’s diagnosis, presentation of clinical symptoms, and access to satisfactory alternative treatment options (e.g., the patient must have a diagnosis of autoantibody positive autoimmune pulmonary alveolar proteinosis 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 treatment
   • The medical team may request additional information from the 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 country laws and regulations about expanded access

Savara will review requests on a case-by-case basis according to the above stated criteria, 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.

Expanded 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 MolgramostimEarlyAccess@Uniphar.com, and a response will be received within approximately 5 business days. All requests must be submitted by a licensed physician. Additional information about expanded access can be found on the Reagan-Udall Foundation Expanded Access Navigator website.