Kedrion Announces Rare Disease Medicine RYPLAZIM® Now Available in US

2024-04-29

上市批准

Distribution resumes of first and only medicine to treat

plasminogen deficiency type 1

FORT LEE, N.J., April 29, 2024 /PRNewswire/ -- Kedrion Biopharma Inc., an international biopharmaceutical company specializing in the research and development, production, and commercialization of plasma-derived therapeutic products used in treating rare and serious diseases, announced today that plasminogen deficiency type 1 (PLGD-1) patients in the U.S. now have access to RYPLAZIM® (plasminogen, human-tvmh).

RYPLAZIM® is the first and only FDA-approved treatment for the approximately 500 people in the U.S. affected by plasminogen deficiency type 1 (PLGD-1). PLGD-1 is an ultra-rare disease that leads to reduced plasminogen activity levels in the body, often resulting in fibrinous lesions on various organs. About 81% of the patients develop lesions on one or both eyes as their lead symptom, but lesions can develop throughout the body on all organs with mucous membranes. Patients with PLGD-1 may require lifelong treatment.

"RYPLAZIM® brings hope and relief to PLGD-1 patients of all ages who have long awaited effective treatment options," said Bob Rossilli, Chief Commercial Officer, Global Business of Kedrion Biopharma. "We are expediting production and distribution to patients and are proud to provide adult and pediatric patients with the first and only FDA-approved treatment designed specifically to address plasminogen deficiency."

The FDA's clearance of RYPLAZIM® sets the stage for Kedrion to pursue additional regulatory approvals in other countries worldwide. It is the latest example of the company's commitment to expanding access to life-changing drugs to rare disease patients globally.

To learn more about RYPLAZIM® and PLGD-1, please visit: ryplazim.com or plasminogen.com

About Kedrion

Kedrion Biopharma is an international biopharmaceutical company specializing in the research and development, production, and commercialization of plasma-derived therapeutic products for use in treating and preventing serious diseases, disorders, and conditions like plasminogen deficiency, hereditary factor X deficiency, and hemophilia. Its portfolio of 37 life-saving products is distributed in over 100 countries.

www.kedrion.com

Contact

Sandy Sklareski

Communications Manager, North America and LATAM

[email protected]

551-255-6557

APPROVED USE

RYPLAZIM® (plasminogen, human-tvmh) is plasma-derived human plasminogen indicated for the treatment of patients with plasminogen deficiency type 1 (hypoplasminogenemia).

IMPORTANT SAFETY INFORMATION

RYPLAZIM is contraindicated in patients with known hypersensitivity to plasminogen or other components of RYPLAZIM.

Do not take RYPLAZIM if you have had a severe reaction to RYPLAZIM or another product containing plasminogen.

Tell your healthcare provider about all of your medical conditions, including any medicines you take, including prescription and non-prescription medicines, such as over-the-counter medicines, supplements or herbal remedies.

RYPLAZIM may worsen or prolong ongoing bleeding. In addition, if you have abnormal tissue growths because of your low plasminogen level, then you may have bleeding as these growths heal in response to treatment with RYPLAZIM.

If you vomit blood, have blood from your rectum or black tarry stools or any bleeding that is heavy or does not resolve after 30 minutes of direct pressure seek emergency care and discontinue further use of RYPLAZIM.

As your lesions heal then they will fall off, known as tissue sloughing, this is in response to treatment with RYPLAZIM. This is also a normal response and is not a side effect. Specific examples:

If you have tissue growths in your airway or lungs you may cough up small amounts of tissue and you may cough up a small amount of blood or blood clots.

If you have tissue growths in your genitourinary system, then you may pass tissue or blood clots in your urine or from your penis or vagina.

If you have tissue growths in your gastrointestinal system you may have some tissue or blood clots in your stool or you may vomit blood or blood clots.

If you have eye lesions you may have oozing of blood from the lesions in your eye.

Notify your healthcare provider if you develop breathing difficulty, wheezing, cough, changes in speech, or pain in the back, abdomen, groin, or pelvic area, because these could be symptoms of tissue from resolving lesions that are obstructing the lungs, the kidneys, the bladder, the uterus and/or vagina, or the intestines.

RYPLAZIM is made from human plasma and therefore carries a risk of transmitting infectious agents, e.g., viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent, and theoretically, the Creutzfeldt-Jakob Disease (CJD) agent.

You may have an allergic reaction while taking RYPLAZIM. Stop treatment and contact your healthcare provider immediately if you develop trouble breathing, wheezing, chest tightness, light headedness, dizziness, swelling in the face or throat, itching, a rash, or hives. Your body may make antibodies against RYPLAZIM that may stop RYPLAZIM from working properly. Your healthcare provider will monitor you periodically for lesions which may be due to a low plasminogen level. If you develop new lesions or a recurrence of a previous lesion, then your healthcare provider will obtain blood to test for plasminogen activity level.

In clinical trials, the most common (incidence ≥ 10%) side effects associated with the infusion of RYPLAZIM were abdominal pain, bloating, nausea, fatigue, extremity pain, hemorrhage, constipation, dry mouth, headache, dizziness, arthralgia, and back pain.

Female patients who are pregnant or may become pregnant should be aware that the potential effects of RYPLAZIM on pregnancy and breastfeeding are unknown. They should notify their physicians if they become or intend to become pregnant, or if they plan to breastfeed.

Other side effects are possible. Speak with your healthcare provider about any side effects that do not go away or bother you.

You are encouraged to report negative side effects of prescription drugs to the FDA. Call 1-800-FDA-1088 or www.fda.gov/medwatch.

For more details, please see full Prescribing Information.

SOURCE Kedrion Biopharma