Purpura, Thrombotic Thrombocytopenic

WEST CHESTER, Ohio, March 5, 2024 /PRNewswire/ -- Technoclone Herstellung von Diagnostika und Arzneimitteln GmbH, world-leading producer of diagnostic tests, equipment, and research products in the field of blood clotting and its exclusive distributor in the USA, DiaPharma Group, Inc., are pleased to announce that the US Food and Drug Administration (FDA) granted De Novo marketing authorization for Technozym ADAMTS13 Activity ELISA. This is the first ADAMTS13 activity test authorized by the FDA. The Technozym ADAMTS13 Activity assay is an enzyme-linked immunosorbent assay (ELISA) intended for the qualitative determination of ADAMTS13 activity in platelet poor human citrated plasma. The assay is intended to be used in conjunction with other clinical and laboratory findings as an aid in the diagnosis of thrombotic thrombocytopenic purpura (TTP) in adult and pediatric patients being evaluated for thrombotic microangiopathy (TMA). "In line with our commitment to provide ADAMTS13 diagnostics globally, we are excited about this milestone, having the first ADAMTS13 activity test granted with marketing authorization by the FDA", says Nikolaus Binder, MD, PhD, CSO of Technoclone. "And today we are honored to address this unmet need for laboratories and patients across the U.S." "We are proud to partner with Technoclone to provide the first in vitro diagnostic assay for ADAMTS13 Activity in the U.S., leading the way for better access to this important parameter", says Madeleine Burmester, President & CEO of DiaPharma. About TTP TTP is a very rare, life-threatening blood clotting disorder caused from either a congenital or acquired absence/decrease of the von Willebrand factor-cleaving protease ADAMTS13. About Technoclone Technoclone is committed to delivering high-quality test systems for in vitro diagnostics in hemostasis to its worldwide clientele. At Technoclone innovation is seen as its responsibility to address emerging complexities in the diagnosis of hematological and cardiovascular disorders. Since 1987, Technoclone has fostered close collaborations with global partners from academia, clinical laboratories, and industry, positioning itself at the forefront of anticipated diagnostic challenges in the years to come. About DiaPharma DiaPharma Group, Inc. sells assay kits, reagents, and instrumentation for laboratory research and clinical applications in the fields of Bleeding and Clotting Disorders and Liver Injury. DiaPharma provides strong personal support and technical competence and experience to ensure customer expectations will be met or exceeded. SOURCE DiaPharma

Thanks to the FDA approval, Takeda said it’s looking to reverse a previous impairment loss tied to Eohilia’s FDA rejection in 2021. Eohilia, an oral suspension formulation of the corticosteroid budesonide, has surprisingly won over the FDA even after the drug’s developer, Takeda, had at one point abandoned the program following an FDA rejection. Monday, Takeda announced the FDA’s approval for Eohilia for treating people 11 years and older with the inflammatory disease eosinophilic esophagitis (EoE). This makes Eohilia the first FDA-sanctioned oral med for the condition. In 2022, Sanofi and Regeneron’s injectable Dupixent claimed the title as the first FDA-approved therapy for EoE. “As the treatment needs and goals of patients with EoE can vary, I welcome the flexibility that Eohilia offers as an oral medication,” Ikuo Hirano, M.D., from Northwestern University, an investigator in Eohilia’s clinical trials, said in a statement Monday. Eosinophilic esophagitis is a chronic inflammatory disease that can cause painful damage to the esophagus, resulting in difficulty in swallowing. The condition affects about 1 in 2,000 people in the U.S., and Takeda has estimated Eohilia could reach $300 million to $500 million in peak sales. Eohilia’s approval comes after some twists and turns. After a regulatory delay caused by additional data submissions, the FDA in late 2021 handed Takeda a rejection, blighting Eohilia’s prospect of becoming the very first official EoE drug. After reviewing the FDA’s complete response letter, Takeda realized that the agency wanted data from an additional clinical trial, and that it could not take the drug any further. “After a deep and careful assessment of the evolving treatment landscape in EoE, as well as considerations including operational challenges required for an additional clinical study, we could not justify further Eohilia development,” Takeda’s R&D chief Andy Plump, M.D., Ph.D., said during an investor call at the time. Fast forward to October 2023, when Takeda suddenly said it had refiled Eohilia in the prior month. This time, Plump said “strong data and remaining unmet need in the U.S. spurred significant grassroots support” for Eohilia from the gastrointestinal and EoE communities. Takeda worked with these communities to include additional data around Eohilia’s benefits in the resubmission, he explained. According to Plump’s comment and Eohilia’s current label, Takeda apparently played down findings from a one-year maintenance study and instead focused on the drug’s 12-week data from a phase 3 and a phase 2 trial in its resubmission. Although that strategy got Eohilia the green light from the FDA, it also led to a restriction. In Eohilia’s label, the FDA notes that the drug hasn’t shown to be effective for longer than 12 weeks. The label later points out that in a phase 3 extension study, continued treatment with Eohilia beyond 12 weeks didn’t show a statistically significant improvement versus re-randomization to placebo on some efficacy endpoints. Still, the drug appears to be a good option for 12 weeks of treatment. Between the phase 3 and phase 2 trials, Eohilia, given at 2mg twice daily, respectively helped 53% and 38% patients achieve histological remission after 12 weeks of treatment, versus 1% and 2.4% for those who received placebo. By comparison, Sanofi and Regeneron won Dupixent’s EoE nod with a 24-week analysis showing the weekly antibody drug led to histological remissions in 60% and 59% of patients in two parts of a phase 3 trial, versus 5% and 6% for the placebo group. As for disease symptoms measured by the patient-reported Dysphagia Symptom Questionnaire (DSQ), Eohilia delivered 10.2- and 14.5-point improvements, compared with 6.5- and 5.9-point improvements for placebo. Results on the same endpoint for Dupixent were 21.9 and 23.8 points, versus 9.6 and 13.9 points for placebo. Thanks to the approval, Takeda said it’s looking to reverse a previously recorded impairment loss tied to Eohilia’s rejection, but that it doesn’t expect the impact to be material. Takeda’s business has come under pressure lately after the Japanese pharma’s blockbuster ADHD drug Vyvanse lost U.S. market exclusivity in August. Adding to the woes are a phase 3 flop for the company’s stem cell therapy Alofisel in complex perianal fistulas, the global market withdrawal of EGFR lung cancer med Exkivity, and the voluntary retraction of an application for the dengue vaccine Qdenga in the U.S. The company has recently won two other FDA nods, one for colorectal cancer drug Fruzaqla, which Takeda in-licensed from Hutchmed a year ago; and the other for rare disease therapy Adzynma in congenital thrombotic thrombocytopenic purpura.

In the aftermath of Vyvanse’s loss of exclusivity, Takeda is making bold pledges to investors to get its business back on track. Despite the recent loss of exclusivity on Takeda's attention-deficit/hyperactivity disorder (ADHD) heavy hitter Vyvanse, the company's portfolio of newer products continues to hold the sales line. During the first nine months of Takeda's 2023 fiscal year, the drugmaker's growth and launch products grew sales 12.7% at constant currencies, Takeda said in a Thursday earnings presentation (PDF). The portfolio accounted for 43% of Takeda’s total sales haul for the period, which ended Dec. 31. Overall, Takeda's global revenues for the nine-month period came to 3.21 trillion Japanese yen (about $22 billion). The number was flat from the prior year at constant exchange rates, but Takeda appears to be taking that performance in stride considering the fact that it’s facing “significant” pressure from Vyvanse generics in the U.S. On that front, Vyvanse sales fell 12.1% to 312.9 billion yen ($2.14 billion), a performance that was “as expected” following the blockbuster’s loss of exclusivity in August. Takeda reports that nine generics to the drug have launched since Aug. 24. Vyvanse's market-share erosion in the U.S. was “slightly milder than initially anticipated,” Takeda said, thanks to constraints on generic supplies of the med. But the generic supply constraints should ease in the coming months, according to Takeda. Meanwhile, Takeda has recently added a pair of new drugs to its launch roster: Fruzaqla, approved in previously treated metastatic colorectal cancer, and Adzynma, an enzyme replacement therapy for congenital thrombotic thrombocytopenic purpura. The stars of Takeda’s drug debut catalogue, however, are its Entyvio pen for inflammatory bowel disease (IBD) and its dengue fever vaccine Qdenga. In recent months, Entyvio maintained its top position in the U.S. IBD market, Takeda said, growing global sales 7% during the first nine months of the company's fiscal year. Entyvio launched in pen form for ulcerative colitis in the U.S. in November. Since then, the drug-device combo has witnessed a “high level of interest and growing formulary access,” Takeda pointed out. Early this year, Takeda hopes to secure a nod for Entyvio in Crohn’s disease. Moving over to Qdenga, the dengue vaccine has now launched in Indonesia, Brazil, Thailand and Argentina. Plus, the shot has experienced “strong initial demand” in private markets, Takeda said. Qdenga has made its way onto some formal government immunization programs, including in Brazil. Elsewhere, Takeda says it’s engaged in “productive discussions” with governments in endemic regions to get the vaccine added to national immunization programs. In the aftermath of Vyvanse’s loss of exclusivity, Takeda is making bold pledges to investors to get its business back on track. In the near term, meaning fiscal year 2024 through 2025, Takeda aims to return to sales, profit and margin growth, the company said in its earnings presentation. Over the medium term, which runs into the early 2030s, Takeda plans to continue expanding the reach of its new medicines. In addition, Takeda hopes to secure more launches from its late-stage pipeline and enjoy “limited” generic competition before the advent of Entyvio biosimilars.