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– Approximately 1,000 patients in the U.S. 12 years of age and older are now eligible for this one-time treatment –
BOSTON, January 16, 2024–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the U.S. Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older.
“Following the historic FDA approval of CASGEVY for the treatment of sickle cell anemia, it is exciting to now obtain DTT approval well ahead of the PDUFA date,” said Reshma Kewalramani, M. D. , CEO and President of Vertex. “TDT patients deserve new options for potentially curative remedies, and we look forward to bringing CASGEVY to eligible patients who are waiting.
Administration of CASGEVY requires mobile stem transplantation; therefore, Vertex is collaborating with revel ind hospitals to identify a network of independent legal remedies (ATCs) in the U. S. CASGEVY will be offered to patients. The nine ATCs activated in the U. S. The U. S. Food and Drug Administration (CDC) may offer CASGEVY to eligible patients with TDT and sickle cell anemia (SCD). Additional ATCs will be activated in the coming weeks and a full list of ATCs can be discovered in CASGEVY. com.
About Beta-Thalassemia Transfusion (TDT)
TDT is a serious and life-threatening genetic disorder. TDT patients report quality of life scores similar to the general population, and lifetime physical care costs in the United States similar to TDT control are estimated to be between $5 and $5. 7 million. TDT requires regular blood transfusions and lifelong iron chelation therapy. Due to anemia, patients living with TDT may experience fatigue and shortness of breath, and babies may develop delayed growth, jaundice, and feeding problems. Complications of TDT can also come with enlarged spleen, liver and/or heart, deformed bones and delayed puberty. DTT requires lifelong treatment and significant use of physical care resources and ultimately results in reduced life expectancy, decreased quality of life, and reduced source of income. and the productivity of life. In the United States, the average age of death for DTT patients is 37 years. Movable stem transplantation from a compatible donor is a curative option, but is only available to a small fraction of people living with TDT due to a lack of available donors.
About CASGEVY™ (exagamglogene autotemcel [exa-cel])
CASGEVY™ is a non-viral, ex vivo, CRISPR/Cas9 gene-edited mobile treatment for eligible patients with SCD or TDT, in which the patient’s hematopoietic stem and mobile progenitors are replaced in the erythroid-specific amplifying region of the gene. BCL11A through exact double strand cutting. This replacement leads to the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in the red blood cells. HbF is the oxygen-carrying form of hemoglobin that naturally provides fetal development, which then transitions to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs in patients with sickle cell anemia and transfusion requirements in patients on DTT.
CASGEVY is approved for certain indications in several jurisdictions for eligible patients.
Helping on your journey CASGEVY
Vertex Connects™ is a program for eligible patients in the U. S. U. S. citizens who have been prescribed CASGEVY. Through Vertex Connects, care managers should provide educational resources, communications, and help walking through the healing process, and more help can be obtained. for eligible patients.
For more information on ATCs, the CASGEVY solution pathway, and Vertex Connects, call 1-833-VERTEX-5 (1-833-837-8395) and learn more about our patient assistance program in VertexConnects. com.
IMPORTANT INFORMATION FOR CASGEVY (exagamglogene autotemcel) in the United States and important safety information
What is CASGEVY?
CASGEVY is an exclusive remedy used to treat other people over the age of 12 who suffer:
• mobile sickle cell anemia (SCD) who have vaso-occlusive crises or common VOCs
• beta-thalassemia (β-thalassemia) requiring blood transfusions
CASGEVY is designed specifically for each patient, uses their own modified blood devices, and increases the production of a special type of hemoglobin called hemoglobin F (fetal hemoglobin or HbF). Having more HbF increases overall hemoglobin levels and has been shown to increase the production and function of red blood cells. This can eliminate VOCs in people with sickle cell anemia and eliminate the need for regular blood transfusions in people with beta-thalassemia.
IMPORTANT SAFETY INFORMATION
What is the most vital information I know about CASGEVY?
After treatment with CASGEVY, you will have fewer blood cells for a while until CASGEVY takes hold (engrafts) into your bone marrow. This includes low levels of platelets (cells that usually help the blood to clot) and white blood cells (cells that usually fight infections). Your doctor will monitor this and give you treatment as required. The doctor will tell you when blood cell levels return to safe levels.
Tell your health care provider right away if you experience any of the following symptoms, which may simply be symptoms of low mobile platelet counts:
severe headache
Abnormal bruising
Prolonged bleeding
bleeding lesions such as nosebleeds; bleeding gums; blood in the urine, stool, or vomit; or coughing up blood
Tell your health care provider right away if you experience any of the following symptoms, which may simply be symptoms of low mobile white blood cell counts:
fever
Chills
Infections
You may experience side effects related to other medications given as part of the CASGEVY treatment regimen. Talk to your doctor about these possible side effects. Your health care provider may prescribe other medications to treat side effects.
How do I get to CASGEVY?
Your health care professional will prescribe other medications, adding a conditioning medication, as a component of your treatment with CASGEVY. It is vital to inform your healthcare professional about the dangers and benefits of all medications involved in your treatment.
After receiving conditioning medicine, you may not be able to get pregnant or have a child. She should discuss fertility preservation features with her healthcare provider before treatment.
STEP 1: Prior to treatment with CASGEVY, a doctor will prescribe one or more mobilization medications. This medication moves blood stem cells from the bone marrow into the bloodstream. The blood stem cells are then collected into a device that separates the other blood cells (this is called apheresis). This total procedure can be performed more than once. Each time, it can take up to a week.
During this stage, rescue cells are also collected and stored in the hospital. These are your existing blood stem cells and are not processed in case there is a challenge in the remedy process. If CASGEVY cannot be given after the conditioning drug, or If the modified blood stem cells do not settle (transplant) into the body, those rescued cells will be returned to you. If you get rescued cells, you won’t get advantages from any healing. Get the benefits of CASGEVY.
STEP 2: After collection, your blood stem cells will be sent to production where they will be used to make CASGEVY. It may take up to 6 months from the time the cells are collected to make and verify CASGEVY before it is returned to your healthcare professional.
STEP 3: Shortly before your stem cell transplant, your healthcare provider will give you a conditioning medicine for a few days in hospital. This will prepare you for treatment by clearing cells from the bone marrow, so they can be replaced with the modified cells in CASGEVY. After you are given this medicine, your blood cell levels will fall to very low levels. You will stay in the hospital for this step and remain in the hospital until after the infusion with CASGEVY.
STEP 4: One or more vials of CASGEVY will be given into a vein (intravenous infusion) over a short period of time.
After the CASGEVY infusion, you will stay in the hospital so that your healthcare provider can closely monitor your recovery. This would possibly take 4-6 weeks, although timelines could vary. Your healthcare provider will let you know when you can go home.
What do I avoid after receiving CASGEVY?
Donate blood, organs, tissues, or cells at any time.
What are the conceivable or rather likely side effects of CASGEVY?
The most common side effects of CASGEVY include:
Low platelet levels, which affects the blood’s ability to clot and cause bleeding.
Low levels of white blood cells, which may make you more susceptible to infection
Your doctor will test your blood for low levels of blood cells (including platelets and white blood cells). Tell your health care provider right away if you experience any of the following symptoms:
fever
Shaking chills
infections
Severe headaches
Abnormal bruising
prolonged bleeding
bleeding lesions such as nosebleeds; bleeding gums; blood in the urine, stool, or vomit; or coughing up blood
These are not all the possible side effects of CASGEVY. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
General Facts About the Effective Use of CASGEVY
Talk to your doctor about any fitness issues.
See the full CASGEVY prescription and patient list.
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including APOL1-mediated kidney disease, acute and neuropathic pain, type 1 diabetes, myotonic dystrophy type 1 and alpha-1 antitrypsin deficiency.
Vertex was founded in 1989 and has its global headquarters in Boston and its foreign headquarters in London. Additionally, the company has studios and development facilities and sales offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently identified as one of the industry’s best employers, scoring 14 consecutive years on Science magazine’s Best Employers list and one of Fortune’s 100 Best Companies to Work For. For corporate updates and more information about Vertex’s history of innovation, visit www. vrtx. com or stay with us on LinkedIn, Facebook, Instagram, YouTube and Twitter/X.
(VRTX-GEN)
Vertex’s Special Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as explained in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements made through Reshma Kewalramani, M. D. , in this press release and statements relating to expectations related to potential benefits. CASGEVY, as it relates to the eligible patient population, plans to engage with hospitals to identify a network of ATCs across the United States and expects additional ATCs to be activated in the coming weeks. Although Vertex believes that the forward-looking statements contained in this press release are accurate, such forward-looking statements constitute the Company’s ideals only as of the date of this press release and there are a number of dangers and uncertainties that may also cause real occasions. or results. be materially different from those expressed or implied by such forward-looking statements. These dangers and uncertainties include, but are not limited to, that knowledge of the Company’s progression systems may not preclude registration or further progression of its compounds for safety, efficacy or other reasons. Obtaining additional approvals and/or commercializing CASGEVY in multiple geographic spaces may not be possible. occur within the expected time frame, or at all, that a good enough price and reimbursement for CASGEVY may not be achieved within the expected time frame, or at all, and other dangers indexed under “Risk Factors” in the annual report of Vertex and in upcoming presentations. with the Securities and Exchange Commission and available on the Company’s website at www. vrtx. com and www. sec. gov. You deserve not to place undue reliance on those statements. Vertex assumes no legal responsibility to update the data contained in this press release as new data becomes available.
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Contacts
Vertex Pharmaceuticals Incorporated Investors: InvestorInfo@vrtx. com or Manisha Pai, 1-617-961-1899 Media: mediainfo@vrtx. com or International: 20 3204 5275 or U. S. U. S. : 617-341-6992 or Heather Nichols: 1-617-839-3607