Dear Hemophilia B Community,

We are writing to share an important update on the availability of HEMGENIX® (etranacogene dezaparvovec-drlb).

CSL is currently experiencing a temporary global stockout of HEMGENIX® that will result in delays in treatment for some individuals in countries with established commercial access.

We want to be very clear that this situation is not related to the safety or effectiveness of HEMGENIX®. Rather, it reflects the complexity of manufacturing gene therapies, and our commitment to adhering to the highest regulatory and quality standards for the people we serve. We are working with regulatory authorities on strategies to ensure stable ongoing supply for HEMGENIX® while preserving our high-quality standards.

We know that this update may raise questions and that’s completely understandable. What we want you to know is that our highest priority, as always, is ensuring that eligible individuals have the information they need to determine if HEMGENIX® is right for them, and that when they do, HEMGENIX® is available in accordance with the highest standards. We remain fully committed to delivering this innovative, one-time gene therapy to the hemophilia B community and continue to have strong confidence in HEMGENIX®.

​We understand the careful thought and planning that go into every individual decision about gene therapy treatment. We deeply value our long‑standing partnership of trust with the hemophilia B community and will continue to keep you updated to ensure people with hemophilia B and their healthcare providers are informed and supported as they plan their hemophilia care needs.

For further information, please visit our contact page on CSL.com here:
https://www.csl.com/contact.
​
Sincerely,
Dr. Deborah Long,
SVP, Medical Affairs

Microhealth, the leading global app in digital hematology, is launching a new digital health
initiative, the PULSE (Period Understanding and Logging of Symptoms for
Empowerment) Pilot in the United States to address a critical gap in bleeding disorders
care: individuals are undertreated due to the normalization of menstrual bleeding, leading
to gaps in access to appropriate treatment and care.

The goal of the PULSE Pilot is to inform a potential expansion of the existing bleed log
within the Microhealth Bleeding Disorders app, specifically to capture menstrual bleeding
and related symptoms more effectively, as well as patient-reported outcomes.
PULSE Pilot volunteers will securely log menstrual cycles and related symptoms with
active consent. Microhealth will collect data in alignment with company data practices,
de-identify and aggregate it, and use it to inform future app development and quality
improvement. This is not a clinical research study. Enrollment is available for individuals in
the United States who menstruate and have a confirmed bleeding disorder diagnosis or are
suspected of having a bleeding disorder. Volunteers can participate regardless of current
treatment or medication status.

For over 15 years, Microhealth Digital Hematology has been an internationally trusted, free
digital tool for the bleeding disorders community worldwide. It lets individuals track bleeds,
symptoms, treatments, physical activity, and health experiences in one place; empowering
them to own their data, advocate for themselves, and optionally connect with Hemophilia
Treatment Centers. The Microhealth Bleeding Disorders app provides care teams with free,
real-time, patient-reported data that reflects their individual lived experience.

With the launch of the PULSE Pilot, Microhealth Digital Hematology aims to make
individuals' menstrual experiences more recognized and valued.

Interested in volunteering for PULSE? Please scan the QR code or visit
https://forms.gle/p8eu9FRV8XZFkMDP6 to join this critical effort.

For further information, please contact Microhealth Community Digital
Advocates Nandini Pethe at [email protected] or Michelle
Cecil at [email protected]. For more information about
Microhealth, visit Microhealth.com.