Avanzanite Bioscience’s Partner Agios Announces PYRUKYND® (mitapivat) Approval in the European Union for Adults with Thalassaemia

AMSTERDAM--(BUSINESS WIRE)--May 22, 2026--

Avanzanite Bioscience B.V., a rapidly growing commercial-stage European specialty pharmaceutical company focused on rare diseases, today reported that its partner, Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company headquartered in Cambridge, Massachusetts focused on delivering innovative medicines for patients with rare diseases, announced that the European Commission has granted marketing authorisation for PYRUKYND® (mitapivat), an oral pyruvate kinase (PK) activator, in adults for the treatment of anaemia associated with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassaemia, with an orphan medicinal product designation.

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“Thalassaemia is a complex, chronic and multisystem disease characterised by anaemia, ineffective erythropoiesis and haemolysis, which together place a significant burden on patients, including a substantial impact on quality of life and persistent fatigue,” commented Raffaella Origa M.D., PhD., Professor of Paediatrics at University of Cagliari, Italy and President of the Italian Society of Thalassaemia and Haemoglobinopathies (SITE). “The approval of PYRUKYND in the EU represents an important step forward, introducing a new oral treatment option regardless of genotype or transfusion burden, with the potential to address key aspects of the disease, including reducing transfusion burden and improving patient outcomes.”

The EC’s decision follows the positive opinion issued by the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) and is based on results from the global, randomised, double-blind, placebo-controlled ENERGIZE and ENERGIZE-T Phase 3 trials. The approval of PYRUKYND for adults with thalassaemia marks its second indication in the EU, following its 2022 approval for adults with PK deficiency.

“The treatment of thalassaemia continues to place a profound burden on patients and healthcare systems,” said Antonis Kattamis M.D., Professor at the National & Kapodistrian University of Athens, Greece, and an investigator in the PYRUKYND thalassaemia Phase 3 clinical program. “An oral therapy such as PYRUKYND has the potential to transform the care of both transfusion-dependent and non-transfusion-dependent patients, and we welcome being able to provide this option in our clinical practice.”

In June 2025, Avanzanite entered into an exclusive agreement with Agios to commercialise and distribute PYRUKYND across the European Economic Area, the United Kingdom, and Switzerland. Avanzanite will continue to work closely with Agios, local health authorities and patient communities to secure access for PYRUKYND across the EU.

“Today’s approval of Agios’ first-in-class PK activator in the EU is great news for adults living with thalassaemia and we are proud and privileged to partner with Agios to distribute and commercialise this medicine in the region,” said Adam Plich, CEO and Co-Founder of Avanzanite Bioscience. “Our role now is to collaborate with local authorities, drive a successful launch, and enable broad access to PYRUKYND in this indication, helping to ensure no thalassaemia patient is left behind, across the EU.”

This milestone represents the fourth rare disease launch that Avanzanite has led, further demonstrating the strength of its pan-European commercial platform. With a ‘Champions League’ team of more than 100 rare disease professionals conducting operations spanning 32 European countries, the company is well-positioned to partner with biotech innovators to deliver transformative therapies to patients across Europe.

About Thalassaemia

Thalassaemia is a rare, inherited blood disease that affects the production of haemoglobin, the protein in red blood cells responsible for carrying oxygen throughout the body. The disease is categorised into two main types: alpha-thalassaemia and beta-thalassaemia, depending on which globin chain of the haemoglobin is affected. By disrupting haemoglobin production, thalassaemia reduces the number of circulating red blood cells and shortens their lifespan, which leads to anaemia, fatigue, and serious complications.

Some individuals with thalassaemia require regular transfusions (classified as transfusion-dependent thalassaemia), while others only need them intermittently (classified as non-transfusion-dependent thalassaemia). All patients with thalassaemia experience a significant disease burden, including comorbidities, reduced quality of life and shortened life expectancy.

About ENERGIZE and ENERGIZE-T

ENERGIZE ( NCT04770753 ) and ENERGIZE-T ( NCT04770779 ) are global, double-blind, placebo-controlled Phase 3 trials evaluating the efficacy and safety of mitapivat in adults with alpha- or beta-thalassaemia.

The ENERGIZE trial randomised 194 non-transfusion-dependent alpha- or beta-thalassaemia patients 2:1 to receive either mitapivat 100 mg twice daily or placebo. The primary endpoint was haemoglobin response, defined as an increase of ≥1.0 g/dL in average haemoglobin concentration from Week 12 through Week 24 compared with baseline. Key secondary endpoints included changes from baseline in average fatigue scores and in average haemoglobin concentration from Week 12 to Week 24. The trial also assessed safety and tolerability.

The ENERGIZE-T trial randomised 258 transfusion-dependent alpha- or beta-thalassaemia patients 2:1 to receive either mitapivat 100 mg twice daily or placebo. The primary endpoint was transfusion reduction response, defined as a ≥50% reduction in transfused red blood cell (RBC) units with a reduction of ≥2 units of RBCs transfused in any consecutive 12-week period through Week 48 compared with baseline. Several transfusion reduction measures were included as key secondary endpoints, and achievement of transfusion independence was a secondary endpoint. The trial also assessed safety and tolerability.

For each trial, patients who completed the double-blind period had the option to transition into a corresponding open-label extension period, during which all patients receive mitapivat.

About Avanzanite Bioscience

Avanzanite is redefining launches of rare disease medicines across Europe. Founded in 2022 and based in Amsterdam, the Netherlands, the company partners with biotech innovators to unlock the full commercial value of orphan medicines through a fully integrated platform spanning 32 countries. With our deep expertise in market access, we navigate Europe’s complex landscape like master chess players – ensuring no patient is left behind while delivering measurable impact and growth opportunities for alliance partners.

For more information, visit www.avanzanite.com.

Adam Plich, CEO and Co-Founder of Avanzanite Bioscience.

KYIV, Ukraine (AP) — Hundreds of Ukrainians marched through the capital on Friday, demanding that the government veto a bill that families of missing soldiers say could lead to their loved ones being prematurely declared dead.

The protesters gathered to oppose Bill No. 13646, which addresses the legal status of missing persons. Participants said that certain provisions of the legislation could allow courts to declare missing Ukrainian military personnel legally dead before their fate has been confirmed.

“Today all the families came out so that the missing are not equated with the dead,” said Mariana Yatselenko, 27, who took part in the Kyiv march.

More than 90,000 people are listed as missing in Ukraine’s unified registry of persons who disappeared under special circumstances, according to Artur Dobrosierdov, the country’s commissioner for missing persons.

Neither Russia nor Ukraine publish regular casualty numbers in the war, although analysts estimate hundreds of thousands of casualties in the fighting.

The Ukrainian register covers people who went missing during combat, as a result of armed aggression or in occupied territories, mostly after Russia’s all-out invasion began on Feb. 24, 2022. But some cases date back to 2014, when Russian soldiers invaded the Crimean Peninsula and pro-Russia forces started fighting in eastern Ukraine.

The registry began operating in May 2023, and at that point, information about both military personnel and civilians from previous years was entered into it.

Similar demonstrations have been held previously over the bill, reflecting growing pressure from relatives of soldiers who are missing.

Ukrainian drones hit a college dormitory building in Starobilsk, a city in Ukraine’s Russia-occupied Luhansk region, killing four people and wounding 39 others, Russian authorities said. Up to 18 people could be buried under the rubble, officials said.

Kremlin spokesman Dmitry Peskov denounced the strike as a “heinous crime.” Ukrainian officials made no immediate comment.

The Russian Defense Ministry on Friday said that it intercepted 217 Ukrainian drones over multiple Russian regions, including the Moscow region and St. Petersburg, the country’s second-largest city.

For the fourth time this month Ukraine struck Russia’s Yaroslavl oil refinery, around 700 kilometers (440 miles) from the border, in an overnight operation, Ukrainian President Volodymyr Zelenskyy said Friday.

Ukraine has been pounding Russian oil facilities in an effort to deny Moscow funding for its invasion.

U.S.-led diplomatic efforts to stop the fighting have brought no significant results and recently appeared to peter out.

“They were not fruitful, unfortunately,” U.S. Secretary of State Marco Rubio said of negotiations over the past year with Russia and Ukraine.

No talks are happening now, he said during a trip to Sweden, although they could resume if Washington sees an opportunity for progress.

Ukraine’s air force said that it shot down or jammed 115 of 124 Russian drones that were launched overnight, in regular bombardments of civilian areas that in recent months have escalated.

Eleven people, including a child, were wounded in Russian attacks across the northern Sumy region, the National Police said. Also, a man was killed by a Russian drone in the city of Kherson, in southern Ukraine, according to the region’s military administration chief.

The number of Ukrainian civilian casualties verified by the United Nations increased by 21% in the first four months of this year, compared with the same period last year, with 815 civilians killed and 4,174 wounded.

In Washington, the Trump administration approved a modest $108 million arms sale to Ukraine that will help the country sustain its midrange air defense missile system.

The U.S. State Department announced the sale of ground-to-air Hawk missile components, spare parts and logistic support late Thursday. Under U.S. President Donald Trump, Washington has slashed military support for Ukraine.

On the battlefield, Ukrainian counterattacks have driven the Russian army out of more than 400 square kilometers (150 square miles) of southern Ukraine since the end of last year, Western analysts say.

Those successes are attributed to Ukraine’s increasingly homegrown drone and missile technology, as well as Russian forces being denied access to Starlink satellite services used to steer drones toward targets.

Zelenskyy said that Russia could be planning new attacks on northern Ukraine, launched from Belarus.

Moscow “is eager to draw (Belarus) deeper into this war,” Zelenskyy said on social media, warning that “there will be consequences” for the Belarusian government, if it provides a platform for strikes on Ukraine.

Ukrainian Foreign Minister Andrii Sybiha alerted allies at a NATO meeting in Sweden about what Ukrainian intelligence services say are growing threats from Belarus. Sybiha urged partners to take unspecified deterrence measures against Minsk.

Russia and Belarus held joint nuclear exercises earlier this week.

The Institute for the Study of War, a Washington-based think tank, underscored “Russia’s ability to leverage Belarus for future Russian military operations and Russia’s deepening de facto control over Belarus.”

Matthew Lee in Washington, and Barry Hatton in Lisbon, Portugal, contributed to this report.

Follow the AP’s coverage of the war in Ukraine at https://apnews.com/hub/russia-ukraine

Women hold a banner with portrait of their relative during a rally of families of missing soldiers in Kyiv, Ukraine, Friday, May 22, 2026. (AP Photo/Efrem Lukatsky)

A woman holds a banner with the portrait of her relative during a rally of families of missing soldiers in Kyiv, Ukraine, Friday, May 22, 2026. (AP Photo/Efrem Lukatsky)

In this image taken from video released by Russian Emergency Ministry Press Service Telegram channel on Friday, May 22, 2026, Rescuers work at the side of a college dormitory building damaged by Ukrainian drones in Starobilsk, Ukraine. (Russian Emergency Ministry Press Service via AP)

A woman looks at a makeshift memorial for fallen soldiers in Russia Ukraine war on Independence Square in Kyiv, Ukraine, Friday, May 22, 2026. (AP Photo/Efrem Lukatsky)