Business Wire

SPEVIGO® granted Breakthrough Therapy Designation in the U.S. for the prevention of generalized pustular psoriasis flares

Share

Boehringer Ingelheim announced today that spesolimab (marketed as SPEVIGO®) received Breakthrough Therapy Designation (BTD) as an investigational treatment for the prevention of flares in adolescents and adults with generalized pustular psoriasis (GPP) from the U.S. Food and Drug Administration (FDA).

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20230502005731/en/

“GPP flares may appear suddenly, intensify quickly, and can be life-threatening if left untreated, leaving those affected feeling anxious and uncertain about their future” said Carinne Brouillon, Member of the Board of Managing Directors and Head of Human Pharma at Boehringer Ingelheim. “The FDA’s recognition of the urgent need for preventing GPP flares is a major step towards empowering people living with the condition to plan critical moments in their lives, despite their disease.”

This designation follows the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA), who also recently awarded spesolimab a BTD for the prevention of GPP flares.

The U.S. FDA and China’s NMPA granted these designations based on the topline results from the EFFISAYIL™ 2 trial which studied whether long-term treatment with the antibody spesolimab helps prevent GPP flares in adolescents and adults with GPP up to 48 weeks.1,2,3 Safety data were in line with previously conducted clinical trials with spesolimab. Data from the trial will be presented at the 25th World Congress of Dermatology 2023 in early July.

About spesolimab
Spesolimab is a novel, humanized, selective antibody that blocks the activation of the interleukin-36 receptor (IL-36R), a signaling pathway within the immune system shown to be involved in the pathogenesis of several autoinflammatory diseases, including GPP.4,6,11 Spesolimab has been approved by regulatory authorities in almost 40 countries including the US, Japan, Mainland China, and the European Union to treat GPP flares in adults.8,9

It is the first approved treatment for GPP flares that specifically targets the IL-36 pathway and that has been evaluated in a statistically powered, randomized, placebo-controlled trial.11 Spesolimab is also under investigation for the treatment of other IL-36 mediated skin diseases.10

About the EFFISAYIL TM clinical trial program
The EFFISAYIL™ clinical trial program includes:

  • EFFISAYIL™ 1: A Phase II study that demonstrated treatment with a single intravenous dose of spesolimab achieved rapid pustular and skin clearance in patients with GPP flares, sustained over 12 weeks.11 These results supported the approval of spesolimab (SPEVIGO®) as the first specific treatment for GPP flares in adults in major markets.8,9,11
  • EFFISAYIL™ 2: A multicenter, randomized, double-blind, placebo-controlled Phase IIb study evaluating the efficacy and safety of maintenance treatment with subcutaneous (SC) spesolimab for the prevention of GPP flares and sustained control of GPP symptoms in adolescents and adults.1,2,3
  • EFFISAYIL™ ON: An open-label, extension study to evaluate the long-term safety and efficacy of spesolimab in patients with GPP, who have completed previous spesolimab trials.5

For the full press release including ‘Notes to Editors’ and references please visit: press release

To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.

Contact information

Boehringer Ingelheim
Corporate Communications
Media + PR

Laura Lessenich
Phone: +49 (6132) 77-173436
Email: press@boehringer-ingelheim.com

Additional information:  www.boehringer-ingelheim.com

About Business Wire

For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.

Subscribe to releases from Business Wire

Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from Business Wire

Deciphera Announces Oral Presentation of Positive Topline Results from Phase 2a Study of Sapablursen in Polycythemia Vera at the 67 th American Society of Hematology (ASH) Annual Meeting6.12.2025 16:30:00 EET | Press release

Deciphera Pharmaceuticals, a member of Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; “Ono”), today announced the oral presentation of positive results from the Phase 2a IMPRSSION study of sapablursen in patients with polycythemia vera (PV) at the 67th American Society of Hematology (ASH) Annual Meeting, taking place December 6-9, 2025, in Orlando, FL. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20251206361611/en/ The results were presented by Ionis Pharmaceuticals, who discovered and developed sapablursen and conducted the IMPRSSION study. In March 2025, Ionis and Ono entered into a license agreement in which Ono obtained exclusive global rights for the development and commercialization of sapablursen. “In the treatment of PV, phlebotomy and cytoreductive therapy are performed as treatments for preventing thrombosis. Phlebotomy is the most common treatment for PV, in which blood

Protagonist and Takeda Present Longer-Term Data at ASH 2025 Showing Rusfertide Delivers Durable Response and Hematocrit Control in Polycythemia Vera6.12.2025 16:30:00 EET | Press release

Protagonist Therapeutics, Inc. (“Protagonist”) (NASDAQ:PTGX) and Takeda (TSE:4502/NYSE:TAK) announce that new 52-week results from the pivotal Phase 3 VERIFY study evaluating rusfertide in patients with polycythemia vera (PV) will be presented in an oral presentation at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition. These findings further reinforce rusfertide’s efficacy and safety and demonstrate durability of response, with 61.9% of patients continuously treated with rusfertide maintaining absence of phlebotomy eligibility from baseline to Week 52. “The 52-week data demonstrated the sustained efficacy of rusfertide, reducing the need for patients to receive phlebotomy while maintaining hematocrit control,” said Dr. Andrew T. Kuykendall, M.D., VERIFY Lead Investigator and Associate Member in the Department of Hematology at Moffitt Cancer Center. “The 32-week VERIFY primary results were already promising, and this deeper understanding of the durability of r

Vertex Presents New Data on CASGEVY ® , Including First-Ever Data in Children Ages 5-11 Years, at the American Society of Hematology Annual Meeting and Announces Plan for Global Regulatory Submissions6.12.2025 14:01:00 EET | Press release

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced data from multiple studies demonstrating the clinical benefits of CASGEVY® (exagamglogene autotemcel) in people ages 5 years and older living with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). The results, including the first presentation of clinical data from pivotal studies in children ages 5-11 years, and longer-term data from the pivotal studies of people with severe SCD and TDT ages 12 years and older, will be presented at the American Society of Hematology (ASH) Annual Meeting. CASGEVY is currently approved for eligible people ages 12 years and older with SCD or TDT in the United States, Great Britain, the European Union, the Kingdom of Saudi Arabia, the Kingdom of Bahrain, Kuwait, Qatar, Canada, Switzerland and the United Arab Emirates. “These results — the first clinical data ever presented on any genetic therapy for children ages 5-11 years with SCD — again demonstrate the tr

Leading Global Scientists Gather at Tengchong Scientists Forum to Explore Innovative Pathways in Frontier Technologies6.12.2025 13:31:00 EET | Press release

The 2025 Tengchong Scientists Forum opened on Saturday in southwest China’s Yunnan Province, convening leading scientists and academic figures to examine innovation across frontier fields including artificial intelligence, biodiversity and quantum technology. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20251205499197/en/ Opening ceremony of Tengchong Scientists Forum on 6th December, 2025 Notable participants include Nobel Physics laureate Konstantin Novoselov, Fields Medalist Efim Zelmanov and Turing Award winner Andrew Chi-Chih Yao, who join 127 academicians, 77 university presidents from China and abroad, over 400 scholars and more than 600 entrepreneurs and financiers. The gathering aims to deepen collaboration between cutting-edge research and industrial development. Under the theme “Science · AI changing the World,” the forum features ten sub-forums, academic sessions and thematic events supporting major cooperation p

Lattice Wins 2025 Global Semiconductor Alliance Award6.12.2025 01:49:00 EET | Press release

Lattice Semiconductor (NASDAQ: LSCC), the low power programmable leader, today announced that it was selected as ‘Most Respected Public Semiconductor Company’ at the 2025 Global Semiconductor Alliance (GSA) Awards. The GSA awards recognize companies that have demonstrated excellence through their success, vision, strategy, and future opportunities in the industry as determined by votes from GSA members. “We are honored to be recognized by the Global Semiconductor Alliance and our peers as one of 2025’s most respected public semiconductor companies. This recognition reflects the dedication of the Lattice team and the trust of our customers, partners, suppliers, and investors. Looking ahead, we remain laser-focused on driving innovation and strengthening our role as the trusted low power programmable leader for semiconductor and system solutions,” said Ford Tamer, Chief Executive Officer, Lattice Semiconductor. The annual GSA Awards celebrate the accomplishments of the semiconductor indu

In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.

Visit our pressroom
World GlobeA line styled icon from Orion Icon Library.HiddenA line styled icon from Orion Icon Library.Eye