Kedrion Biopharma to commercialize RYPLAZIM® in U.S

  • RYPLAZIM is indicated to treat plasminogen deficiency type 1, also known as C-PLGD, an ultra-rare condition affecting less than 2,000 people in the U.S. 1
  • C-PLGD is characterized by lesions that can lead to blindness, respiratory failure and other severe complications
  • C-PLGD impacts infants and children most severely
  • RYPLAZIM received FDA approval in June, 2021; prior to that approval, it was available only on a compassionate use basis in the U.S.
  • Kedrion Biopharma is now ramping up for a 2022 U.S. launch of RYPLAZIM, which was added to the Company’s U.S. commercial portfolio as a result of Kedrion Biopharma acquiring Prometic BioTherapeutics Inc. last week; the deal further strengthens the Company’s global leadership position in the manufacture, commercialization and distribution of plasma-derived therapies used in treating certain rare and serious conditions.

Fort Lee, NJ (October 20, 2021) – Kedrion Biopharma, an international biopharmaceutical company specialized in the manufacture and distribution of plasma-derived therapeutic products used in treating rare and serious diseases, is now gearing up to commercialize and distribute RYPLAZIM® (plasminogen human-tvmh) in the United States. RYPLAZIM is indicated to treat plasminogen deficiency type 1, also known as C-PLGD, an ultra-rare condition affecting less than 2,000 people in the U.S. 1

A lifelong disease, the most severe symptoms of C-PLGD are observed in infants and children.1 Given its rarity, this condition is probably underdiagnosed in the U.S.

As announced earlier, Kedrion Biopharma has completed acquisition of the Prometic life sciences business in North America, further strengthening its global leadership position in the manufacture, commercialization and distribution of plasma-derived therapies used in treating certain rare and serious conditions.

 “The most important mission at Kedrion Biopharma is to improve the lives of people with rare and serious diseases,” said Kedrion Biopharma Chief Executive Officer, Val Romberg. “As the newest addition to our growing portfolio of products, RYPLAZIM is an excellent example of that dedication. RYPLAZIM meets an urgent unmet medical need for people who face plasminogen deficiency type 1, a potentially devastating, but treatable, medical condition. We are pleased and gratified to be in a position now to help these patients.”

With the acquisition of Prometic BioTherapeutics Inc., Kedrion finalized a series of transactions that began last June with the announcement that it was acquiring from the Nasdaq-quoted Liminal Biosciences Inc., the plasma purification plant at Laval in Quebec, and the license to distribute the new product in the United States. The Prometic deal also brings more cutting-edge purification technology to the Kedrion Biopharma group.

Individuals with C-PLGD lack plasminogen, a protein that enables the body to break down fibrin clots, which are involved in the final stages of blood clotting in humans.2 This results in accumulation of fibrin and finally leads to development of lesions which, in about 81 percent of patients, affect the eyes in a condition known as ligneous conjunctivitis.1 Blindness can be a consequence.3 Lesions as a consequence of plasminogen deficiency can also affect the mouth in approximately 30 percent of patients in a condition known as ligneous gingivitis. About 20 percent of patients experience life-threatening airway obstruction. Several other organ systems may be impacted by C-PLGD, as well.1

RYPLAZIM is plasminogen purified from human plasma, administered intravenously. The treatment helps to increase the plasma level of plasminogen, enabling a temporary correction of the plasminogen deficiency and reduction or resolution of the lesions. In a single-arm, open-label clinical trial (N=15 patients) the overall rate of clinical success was met as all patents with any lesion at baseline had at least 50 percent improvement in the number/size of their lesions. The study also showed that 78 percent of all external lesions and 75 percent of all internal lesions were resolved by the end of week 48. And there were no recurrent or new external or internal lesions in any patient through week 48.4

Kedrion Biopharma is ramping up RYPLAZIM production capacity to meet patient demand and anticipates RYPLAZIM will be available in limited quantities in 2022.

RYPLAZIM®

Cautionary Statement Regarding Forward-Looking Statements:

This press release contains “forward looking statements” within the meaningof the Private Securities Litigation Reform Act of 1995 regarding, amongother things, the research, development and commercialization ofpharmaceutical products. All statements that are not statements of historicalfacts are, or may be deemed to be, forward-looking statements. Suchforward-looking statements are based on historical performance and currentexpectations and projections about our future financial results, goals, plansand objectives and involve inherent risks, assumptions and uncertainties,including internal or external factors that could delay, divert or change any ofthem in the next several years, that are difficult to predict, may be beyond ourcontrol and could cause our future financial results, goals, plans andobjectives to differ materially from those expressed in, or implied by, thestatements. These risks, assumptions, uncertainties and other factorsinclude, among others, the possibility of unfavorable results from furtherclinical trials involving the product candidate. Forward-looking statements inthis press release should be evaluated together with the many risks anduncertainties that affect Kedrion Biopharma’s business andmarket, particularly those identified in the cautionarystatement and risk factors discussion in Kedrion Biopharma’sAnnual Report on Form 10-K for the year ending December31, 2020, as updated by our subsequent Quarterly Reports onForm 10-Q, Current Reports on Form 8-K and other filingswith the Securities and Exchange Commission. The forward-lookingstatements included in this document are made onlyas of the date of this document and except as otherwiserequired by applicable law, Kedrion Biopharma undertakes noobligation to publicly update or revise any forward-lookingstatement, whether as a result of new information, futureevents, changed circumstances or otherwise.

About RYPLAZIM®

INDICATIONS AND USAGE

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

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS:

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

WARNINGS AND PRECAUTIONS:

  • Bleeding: RYPLAZIM administration may lead to bleeding at active mucosal disease-related lesion sites or worsen active bleeding not related to disease lesions. Discontinue RYPLAZIM if serious bleeding occurs. Monitor patients during and for 4 hours after infusion when administering RYPLAZIM to patients with bleeding diatheses and patients taking anticoagulants, antiplatelet drugs, or other agents which may interfere with normal coagulation.
  • Tissue Sloughing: Respiratory distress due to tissue sloughing may occur in patients with mucosal lesions in the tracheobronchial tree following RYPLAZIM administration. Please monitor appropriately.
  • Transmission of Infectious Agents: 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.
  • Hypersensitivity Reactions: Hypersensitivity reactions, including anaphylaxis, may occur with RYPLAZIM. If symptoms occur, discontinue RYPLAZIM and administer appropriate treatment.
  • Neutralizing Antibodies: Neutralizing antibodies (inhibitors) may develop, although they were not observed in clinical trials. If clinical efficacy is not maintained (e.g., development of new or recurrent lesions), determine plasminogen activity trough levels in plasma.
  • Laboratory Abnormalities: Patients receiving RYPLAZIM may have elevated blood levels of D-dimer. D-dimer levels will lack interpretability in patients being screened for venous thromboembolism (VTE).

ADVERSE REACTIONS:

The most frequent (incidence ≥ 10 percent) adverse reactions in clinical trials were abdominal pain, bloating, nausea, fatigue, extremity pain, hemorrhage, constipation, dry mouth, headache, dizziness, arthralgia, and back pain.

To report SUSPECTED ADVERSE REACTIONS, contact KEDRION at 1-855-427-6378 or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch

Please see RYPLAZIM full Prescribing Information for complete prescribing details. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. 

About Kedrion Biopharma

Kedrion Biopharma is a leading international biopharmaceutical company that specializes in the development, production and distribution of plasma-derived therapeutic products for use in treating serious diseases, disorders and conditions such as immune system deficiencies and coagulation disorders. Kedrion has special expertise in the treatment of rare diseases and in the manufacture of hyper-immune products such as anti-hepatitis B, anti-tetanus and RhoGAM® for prophylaxis against Rh sensitization and subsequent hemolytic disease of the fetus and newborn. The company operates through a fully integrated business model from the collection of plasma in its own centers in North America to fractionation and production in its manufacturing facilities located in Italy, Hungary and North America.

Headquartered in Castelvecchio Pascoli (Italy), Kedrion has over 2,700 employees and a commercial presence in approximately 100 countries worldwide. Kedrion places a high value on the welfare of those who benefit from its products, as well as on the people and the communities it serves. Additional information about Kedrion Biopharma can be found at kedrion.com and kedrion.us.

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CONTACT:

Kedrion Biopharma: Sheila A. Burke Method Health Communications 484-667-6330 [email protected]           

References:

1.     An international registry of patients with plasminogen deficiency, (HISTORY). Shapiro et al. Jan 30, 2020.    https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7049368/. Accessed 10/12/21

2.     Arteriosclerosis, Thrombosis, and Vascular Biology, Vol. 31, No. 12, Fibrin Clot Structure and Function. Undas     et al. https://www.ahajournals.org/doi/full/10.1161/atvbaha.111.230631. Accessed 10/12/21

3.     New England Journal of Medicine. Schott et al. Jan 3, 1998.

        https://www.nejm.org/doi/full/10.1056/nejm199812033392305. Accessed 10/12/21

4. Plasminogen replacement therapy for the treatment of children and adults with congenital plasminogen deficiency. Shapiro et al. BLOOD. March 22, 2018. Accessed 10/13/21.



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