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Business Wire
7 days ago
- Health
- Business Wire
KalVista Pharmaceuticals Receives Positive CHMP Opinion for Sebetralstat for the Treatment of Hereditary Angioedema Attacks
CAMBRIDGE, Mass. & SALISBURY, England--(BUSINESS WIRE)--KalVista Pharmaceuticals, Inc. (Nasdaq: KALV) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending marketing authorization for sebetralstat, a novel oral plasma kallikrein inhibitor, for symptomatic treatment of acute attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older. The European Commission (EC) final decision is expected by early October. 'This positive CHMP opinion is an important step forward for people living with HAE in Europe, where there remains a high need for effective, easy-to-administer on-demand treatments,' said Ben Palleiko, CEO of KalVista. 'Coming just weeks after regulatory approvals in the US and UK, this milestone highlights the potential of sebetralstat to transform how HAE is managed globally. Sebetralstat is expected to become the first and only oral on-demand treatment for HAE available in Europe, bringing forward a new treatment approach that enables adherence to guidelines and empowers people to treat attacks quickly, wherever they occur.' The CHMP based its opinion on results from the phase 3 KONFIDENT clinical trial, which was the largest clinical trial ever conducted in HAE. Data from KONFIDENT was published in the New England Journal of Medicine in May 2024, showing that sebetralstat achieved significantly faster symptom relief, reduction in attack severity and attack resolution than placebo, and was well-tolerated with a safety profile similar to placebo.1 The trial randomized 136 HAE patients from 66 clinical sites across 20 countries. 'Access to on-demand treatments that offer easy self-administration and rapid symptom relief is critical for patients living with HAE,' said Emel Aygören-Pürsün, M.D., Assistant Professor, Internal Medicine and Hemostaseology, Department for Children and Adolescents, University Hospital Frankfurt. 'Until now, on-demand therapies for HAE attacks required parenteral administration via injections. The positive CHMP opinion for sebetralstat is an essential move toward a novel, long-awaited oral on-demand option that could help patients treat attacks early and independently, as advocated by guidelines, with the goal of improving outcomes and normalizing lives.' Sebetralstat is approved in the United States and United Kingdom under the brand name EKTERLY® for the treatment of HAE attacks in people 12 years of age and older. It is currently under review with regulatory authorities in Japan as well as multiple other territories. About Hereditary Angioedema Hereditary angioedema (HAE) is a rare genetic disease resulting in deficiency or dysfunction in the C1 esterase inhibitor (C1INH) protein and subsequent uncontrolled activation of the kallikrein-kinin system. People living with HAE experience painful and debilitating attacks of tissue swelling in various locations of the body that can be life-threatening depending on the area affected. Treatment guidelines recommend treating attacks as early as possible to prevent progression of swelling and shorten the time to attack resolution, and to consider treatment for all attacks, regardless of anatomic location or severity. About Sebetralstat Sebetralstat is a novel plasma kallikrein inhibitor approved in the United States and United Kingdom for the treatment of acute attacks of hereditary angioedema (HAE) in people 12 years of age and older. It is the first and only oral on-demand treatment for HAE. With ongoing studies exploring its use in children aged two to 11 and multiple regulatory applications under review in key global markets, sebetralstat has the potential to become the foundational therapy for HAE management worldwide. U.S. INDICATION AND IMPORTANT SAFETY INFORMATION What is EKTERLY® (sebetralstat)? EKTERLY is a prescription medicine used to treat sudden (acute) attacks of hereditary angioedema (HAE) in adults and children aged 12 years of age and older. It is not known if EKTERLY is safe and effective in children under 12 years of age. IMPORTANT SAFETY INFORMATION Before taking EKTERLY, tell your healthcare provider about all of your medical conditions, including if you: Are pregnant or planning to become pregnant. It is not known if EKTERLY can harm your unborn baby. Are breastfeeding or plan to breastfeed. It is not known if EKTERLY passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby while taking EKTERLY. Have liver problems. Tell your healthcare provider about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking EKTERLY with certain other medicines can cause side effects or affect how well EKTERLY or the other medicines work. Especially tell your healthcare provider if you take any of the following, as their use with EKTERLY is not recommended: itraconazole, phenytoin, efavirenz. Know the medicines you take. Keep a list of them to show your healthcare provider or pharmacist when you get a new medicine. What are the possible side effects of EKTERLY? The most common side effects of EKTERLY include headache. For more information, ask your healthcare provider or pharmacist. Talk to your doctor for medical advice about side effects. You are encouraged to report side effects related to KalVista products by calling 1-855-258-4782. If you prefer, you may contact the U.S. Food and Drug Administration (FDA) directly. Visit or call 1-800-FDA-1088. Please click here for full Prescribing Information, including Patient Information. About KalVista Pharmaceuticals, Inc. KalVista Pharmaceuticals, Inc., is a global biopharmaceutical company dedicated to developing and delivering life-changing oral therapies for individuals affected by rare diseases with significant unmet needs. In the U.S., KalVista markets EKTERLY®, the first and only oral on-demand treatment for hereditary angioedema (HAE). The Company has multiple regulatory applications under review in key global markets. For more information about KalVista, please visit and follow us on LinkedIn, X, Facebook and Instagram. Forward-Looking Statements This press release contains "forward-looking" statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: "anticipate," "intend," "plan," "goal," "seek," "believe," "project," "estimate," "expect," "strategy," "future," "likely," "may," "should," "will" and similar references to future periods. These statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from what we expect. Examples of forward-looking statements include, among others, information relating to our business and business plans, the success of our efforts to commercialize EKTERLY® (sebetralstat), our ability to successfully obtain foreign regulatory approvals for sebetralstat, including approval by the EC, our expectations about the timing of the EC's final decision, our expectations about the safety and efficacy of sebetralstat and our other product candidates, the timing of clinical trials and their results, our ability to commence clinical studies or complete ongoing clinical studies, including our KONFIDENT-S and KONFIDENT-KID trials, and the ability of EKTERLY to treat HAE. Further information on potential risk factors that could affect our business and financial results are detailed in our filings with the Securities and Exchange Commission, including in our annual report on Form 10-K for the year ended April 30, 2025, our quarterly reports on Form 10-Q, and our other reports that we may make from time to time with the Securities and Exchange Commission. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise. _______________________ Expand 1Riedl MA, et al. Oral sebetralstat for on-demand treatment of hereditary angioedema attacks. N Engl J Med. 2024;391(1):32–43. Expand
Business Wire
15-07-2025
- Business
- Business Wire
KalVista Pharmaceuticals Announces UK MHRA Approval of EKTERLY ® (sebetralstat), First and Only Oral On-demand Treatment for Hereditary Angioedema
CAMBRIDGE, Mass. & SALISBURY, England--(BUSINESS WIRE)-- KalVista Pharmaceuticals, Inc. (Nasdaq: KALV) today announced that the Medicines and Healthcare products Regulatory Agency (MHRA) of the United Kingdom (UK) has granted marketing authorization for EKTERLY ® (sebetralstat), a novel plasma kallikrein inhibitor, for the treatment of hereditary angioedema (HAE) attacks in adults and adolescents aged 12 years and older. EKTERLY is the first and only oral on-demand treatment for HAE approved in the UK. 'The approval of EKTERLY in the United Kingdom builds on our recent FDA approval and advances our global commitment to bring this innovative treatment option to people living with HAE,' said Ben Palleiko, CEO of KalVista. 'This approval holds particular significance for KalVista as EKTERLY was discovered in our Salisbury, UK labs—making it even more exciting that now we will be able to offer this therapy to people living with HAE in the UK. More broadly, this is another example of the incredible skills of our entire team in pursuing what we expect to be the fastest global launch of an HAE therapy ever.' The MHRA's marketing authorization for EKTERLY is based on results from the phase 3 KONFIDENT clinical trial, which was the largest clinical study ever conducted in HAE. Data from KONFIDENT was published in the New England Journal of Medicine in May 2024, showing that EKTERLY achieved significantly faster symptom relief, reduction in attack severity and attack resolution than placebo, and was well-tolerated with a safety profile similar to placebo. The trial randomized 136 HAE patients from 66 clinical sites across 20 countries. 'The MHRA approval of EKTERLY brings us one step closer to having an oral on-demand treatment available to patients in the UK for the first time,' said Dr. Sinisa Savic, Professor of Clinical Immunology, St James's University Hospital Leeds and sebetralstat investigator. 'I look forward to seeing the impact this new treatment option will have on the HAE community in the UK.' EKTERLY also met the requirements of the MHRA Orphan Designation criteria and will be added to the Orphan Register held by the MHRA, allowing it to benefit from up to 10 years of market exclusivity. In March of this year, sebetralstat was granted a positive scientific opinion for an Early Access to Medicines Scheme (EAMS) from the MHRA for the treatment of HAE attacks. This has enabled healthcare professionals to prescribe the treatment prior to marketing authorization, based on clinical factors for patients with a clear unmet need. It will continue to be available through the EAMS until KalVista receives a decision from the National Institute for Health and Care Excellence (NICE) for use of EKTERLY under the UK's National Health Service (NHS), which is anticipated in the first half of 2026. On July 3, 2025, EKTERLY was approved by the U.S. Food and Drug Administration for the treatment of HAE attacks in people 12 years of age and older. KalVista has submitted marketing authorization applications for sebetralstat in the EU, Japan and other key global markets. About Hereditary Angioedema Hereditary angioedema (HAE) is a rare genetic disease resulting in deficiency or dysfunction in the C1 esterase inhibitor (C1INH) protein and subsequent uncontrolled activation of the kallikrein-kinin system. People living with HAE experience painful and debilitating attacks of tissue swelling in various locations of the body that can be life-threatening depending on the area affected. Treatment guidelines recommend treating attacks as early as possible to prevent progression of swelling and shorten the time to attack resolution, and to consider treatment for all attacks, regardless of anatomic location or severity. About EKTERLY ® (sebetralstat) EKTERLY (sebetralstat), a novel plasma kallikrein inhibitor, is the first and only oral on-demand treatment for hereditary angioedema (HAE) attacks in adults and adolescents aged 12 years and older. With ongoing studies exploring its use in children aged two to 11 and multiple regulatory applications under review in key global markets, EKTERLY has the potential to become the foundational therapy for HAE management worldwide. For complete product information, please see the Summary of Product Characteristics that can be found on About KalVista Pharmaceuticals, Inc. KalVista Pharmaceuticals, Inc., is a global biopharmaceutical company dedicated to developing and delivering life-changing oral therapies for individuals affected by rare diseases with significant unmet needs. In the U.S., KalVista markets EKTERLY ®, the first and only oral on-demand treatment for hereditary angioedema (HAE). The Company has multiple regulatory applications under review in key global markets. For more information about KalVista, please visit and follow us on LinkedIn, X, Facebook and Instagram. Forward-Looking Statements This press release contains "forward-looking" statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: "anticipate," "intend," "plan," "goal," "seek," "believe," "project," "estimate," "expect," "strategy," "future," "likely," "may," "should," "will" and similar references to future periods. These statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from what we expect. Examples of forward-looking statements include, among others, information relating to our business and business plans, the success of our efforts to commercialize EKTERLY ® (sebetralstat), our ability to successfully obtain foreign regulatory approvals for sebetralstat, including the outcome of the decision from the NICE regarding the use of EKTERLY under the UK's NHS, our expectations about the safety and efficacy of sebetralstat and our other product candidates, the timing of clinical trials and their results, our ability to commence clinical studies or complete ongoing clinical studies, including our KONFIDENT-S and KONFIDENT-KID trials, and the ability of EKTERLY to treat HAE , and the future progress and potential success of our oral Factor XIIa program. Further information on potential risk factors that could affect our business and financial results are detailed in our filings with the Securities and Exchange Commission, including in our annual report on Form 10-K for the year ended April 30, 2025, our quarterly reports on Form 10-Q, and our other reports that we may make from time to time with the Securities and Exchange Commission. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.
WebMD
08-07-2025
- Health
- WebMD
FDA Approves First Oral Treatment for Rare Swelling Disorder
July 8, 2025 – The FDA has approved the first-ever pill for a rare genetic swelling disorder. The drug, known as sebetralstat but sold as Ekterly, is for adults and children age 12 years or older with hereditary angioedema (HAE), which affects about 1 in 50,000 people and causes sudden and painful swelling that can sometimes be life-threatening. These easy-to-take pills help stop an attack whenever it happens, offering an advantage over other treatments that need to be injected either into the veins or under the skin. HAE happens when a person has a faulty gene that causes their body to make too little of a protein called C1 inhibitor, or the protein doesn't work well. This protein usually helps control swelling and inflammation in the body. This genetic defect can cause swelling in the face, throat, abdomen, or limbs, which can sometimes be serious. Those with the condition who have swelling in the abdomen can have stomach pain, vomiting, or diarrhea. Swelling in the throat or face can make it hard to breathe. Even with regular treatment, many people with HAE still have sudden attacks and always need fast-acting medicine available to them. "As the first orally administered on-demand therapy for HAE attacks, Ekterly provides patients and physicians with an important and welcome advance in HAE treatment options," Anthony J. Castaldo, chief executive officer of the US Hereditary Angioedema Association, said in a statement. Ekterly was approved after a study involving 136 people with HAE across 20 countries showed that it could quickly relieve symptoms and help end attacks sooner, compared to a placebo, regardless of how severe the attack was. In a follow-up study, many people were able to take the pill within 10 minutes of an attack, and symptoms began to improve in about 1.3 hours, according to a news release from KalVista Pharmaceuticals, the drug's maker. Ekterly works by blocking an enzyme called plasma kallikrein, which causes swelling. In people with HAE, this enzyme is too active because the C1 inhibitor protein does not work properly. By blocking this enzyme, Ekterly helps stop sudden swelling and pain from happening. The pill lets people treat attacks early, giving them more control and helping doctors lower the impact of the disease. According to KalVista, the medicine will be available right away. Headaches are the only reported side effect of the new treatment. It's not known if the medicine is safe during pregnancy or breastfeeding, so patients should tell their doctor if they are pregnant, planning to become pregnant, or breastfeeding. Patients should also tell their doctor if they have liver problems or are taking other medicines, especially for fungal or viral infections, or epilepsy.
Cision Canada
08-07-2025
- Business
- Cision Canada
DRI Healthcare Comments on FDA Approval and our Increased Investment in KalVista Pharmaceuticals' Ekterly® (sebetralstat)
– Ekterly is the first and only oral on-demand therapy for treating attacks associated with hereditary angioedema – – DRI Healthcare is entitled to a tiered royalty on worldwide net sales of Ekterly – – KalVista has elected to receive additional funding increasing our total investment to $127 million – TORONTO, July 8, 2025 /CNW/ - DRI Healthcare Trust (TSX: (TSX: DHT.U) ("DRI Healthcare") today announced KalVista Pharmaceuticals ("KalVista") has disclosed it has received approval from the U.S. Food and Drug Administration ("FDA") for Ekterly (sebetralstat). Ekterly is approved for the treatment of acute attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older. HAE is a rare genetic disorder characterized by recurring episodes of severe swelling in various parts of the body, including the face, extremities, gastrointestinal tract, and airways. "We are very pleased that the FDA has approved Ekterly as the first and only oral on-demand therapy for treating HAE attacks," said Ali Hedayat, Chief Executive Officer of DRI Healthcare. "The addition of the long-dated Ekterly cash flows demonstrates the value that a pre-approval transaction can bring to our portfolio." "We congratulate our partners at KalVista, who worked diligently to develop Ekterly to help transform the lives of patients who suffer from HAE," added Navin Jacob, Executive Vice President & Chief Investment Officer. KalVista has notified DRI Healthcare that it has elected to receive the optional payment of $22 million as part of the November 2024 royalty transaction. As a result of receiving this one-time payment, the royalty rate on the first sales tranche steps up and the sales-based milestone amount increases. The transaction now entitles DRI Healthcare to a tiered royalty of 6.00% on net sales up to and including $500 million, 1.10% on net sales above $500 million and up to and including $750 million, and 0.25% on net sales above $750 million. KalVista is entitled to a potential one-time sales-based milestone payment of $57 million if annual worldwide net sales of sebetralstat meet or exceed $550 million in any calendar year before January 1, 2031. About DRI Healthcare DRI Healthcare is a pioneer in global pharmaceutical royalty monetization. Since our founding in 1989, we have deployed more than $3.0 billion, acquiring more than 75 royalties on 45-plus drugs, including Ekterly, Eylea, Keytruda, Orserdu, Remicade, Spinraza, Stelara, Vonjo and Zytiga. DRI Healthcare's units are listed and trade on the Toronto Stock Exchange in Canadian dollars under the symbol " and in U.S. dollars under the symbol "DHT.U". To learn more, visit or follow us on LinkedIn.
Yahoo
07-07-2025
- Health
- Yahoo
Orsini Selected as Specialty Pharmacy Partner for KalVista's EKTERLY® (sebetralstat)
EKTERLY is an FDA-approved oral therapy for the on-demand treatment of hereditary angioedema (HAE) ELK GROVE VILLAGE, Ill., July 7, 2025 /PRNewswire/ -- Orsini, a leader in rare disease pharmacy solutions, has been chosen by KalVista Pharmaceuticals as a specialty pharmacy partner for EKTERLY® (sepiapterin), an FDA-approved oral therapy for the treatment of acute attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older. Read the full Prescribing Information here. HAE is a life-threatening inherited genetic disorder that can affect the autoimmune system, causing recurrent episodes of severe swelling, most commonly in the limbs, face, intestines and airway. The condition is a result of low levels or improper functioning of a protein called C1 inhibitor. While minor trauma or stress could trigger an attack, swelling often occurs without a known trigger. EKTERLY is administered at the onset of an HAE attack to quickly relieve symptoms and reduce the severity and duration of the episode. It is the tenth HAE therapy to join Orsini's portfolio. "Orsini has been a leader in the HAE space for over 14 years, and we are deeply committed to giving patients the care they need to overcome the day-to-day struggles they face," Darin DeCarlo, Orsini's Chief Commercial Officer, said. "It's our mission to ensure that no patient gets left behind, so we're thrilled to have another way to serve the HAE community with EKTERLY." About OrsiniProviding patients with comprehensive and compassionate care since 1987, Orsini is a leader in rare diseases and gene therapies. Orsini partners with biopharma innovators, healthcare providers and payors to support patients and their families in accessing revolutionary treatments for rare diseases. Through integrated rare disease pharmacy solutions including pharmacy distribution, patient services, clinical management and convenient home infusion services, Orsini simplifies how patients connect to advanced therapies. Orsini's high-touch care model centers on experienced and trained therapy care teams that provide personalized patient care to ensure that No Patient is Left Behind™. Orsini holds accreditations with the Accreditation Commission for Health Care (ACHC), The Joint Commission, URAC and NABP. Orsini has earned URAC's Rare Disease Pharmacy Center of Excellence Designation and ACHC's Distinction in Rare Diseases and Orphan Drugs. For more information, visit View original content to download multimedia: SOURCE Orsini Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
