The EU committee’s negative opinion for Elevidys is yet another major setback for the company

International News via Reuters:

Sarepta Therapeutics failed to win the European drug regulator’s backing for its muscle disorder gene therapy on Friday, as the company faces intense regulatory scrutiny after two recent patient deaths tied to the treatment.

Shares of the Cambridge, Massachusetts-based company slumped 11%. They have lost 88.8% so far this year.

The EU committee’s negative opinion for Elevidys is yet another major setback for the company, which was directed by the U.S. health regulator earlier this month to stop all shipments of the gene therapy — the only one approved by it for treating Duchenne muscular dystrophy (DMD).

Sarepta had already halted dosing in non-ambulatory patients, meaning those unable to walk independently, and later complied with the U.S. Food and Drug Administration’s request to pause all shipments, along with Roche — its partner outside the U.S.

“While we are disappointed by CHMP’s (Committee for Medicinal Products for Human Use) negative opinion, we understand the urgent need for continued dialogue and collaboration to bring transformative therapies to people with Duchenne,” said Sarepta’s research and development head, Louise Rodino-Klapac.

DMD is primarily seen in males, affecting 1 in 5,000 boys born worldwide. The average life expectancy for someone with the disorder is 22 years.

William Blair analyst Sami Corwin said the fall in Sarepta shares reflected the reduced revenue from potential future milestone and royalty payments from Roche.

“While we believe there is a possibility the pause on Elevidys shipments in the U.S. is lifted before year-end specifically for young and ambulatory patients… the uncertainty will continue to pressure stock.”

Roche said on Friday it would continue its dialogue with the European Medicines Agency to explore a potential path forward to make Elevidys available in the EU.

The benefit-risk profile remains positive in the ambulatory patients, the Swiss drugmaker said.

Sarepta said it continues to work with the FDA in the U.S. to address recent safety concerns related to Elevidys.

Reporting by Christy Santhosh in Bengaluru; Editing by Shilpi Majumdar

The inhaler, with an older propellant, is already approved in Europe for the treatment of adults with chronic obstructive pulmonary disease

International News via Reuters:

A panel of the European medicines regulator has recommended approving an eco-friendly version of AstraZeneca’s inhaler for the treatment of a chronic lung condition, the drugmaker said on Friday.

The European Medicines Agency’s Committee for Medicinal Products for Human Use has backed the use of a new type of a pressurised gas in the company’s triple-drug inhaler Trixeo Aerosphere.

In this version, Trixeo Aerosphere, also called Breztri Aerosphere in some regions, will use a next-generation, medical-grade propellant from Honeywell to deliver metered-doses to patients’ lungs as part of AstraZeneca’s green push.

The inhaler, with an older propellant, is already approved in Europe for the treatment of adults with chronic obstructive pulmonary disease, commonly referred to “smoker’s lung” as the disease typically affects cigarette smokers. But it can also be caused by air pollution and related occupational hazards.

Trixeo Aerosphere’s eco-friendly version was approved by British regulators in May, marking the world’s first, and is currently under review in other countries, including China.

CHMP’s recommendation for European Union’s approval is based on the results from trials, which showed the latest propellant was comparable to the current one in delivering doses, without any new safety risks.

“CHMP’s positive opinion of Trixeo Aerosphere with the next-generation propellant allows us to address the needs of both patients and the planet,” AstraZeneca executive Ruud Dobber said.

Honeywell’s new propellant offers up to 99.9% lower global warming potential compared to those currently used, leaving a lower carbon footprint.

If formally approved by the European Commission, which has the final authority, Trixeo Aerosphere would be the first in the EU to use the new delivery mechanism. The EC typically follows the advise of the EMA, but is not bound to do so.

The inhaler made $978 million in revenue for AstraZeneca in 2024. Rival therapies for COPD include Sanofi-Regeneron’s Dupixent, GSK’s Trelegy Ellipta and Verona Pharma’s Ohtuvayre.

AstraZeneca said it would now begin to transition its Trixeo supply to the new propellant in Europe, based on CHMP’s backing.

Reporting by Pushkala Aripaka and Unnamalai L in Bengaluru; Editing by Shilpi Majumdar

The ECB left interest rates unchanged on Thursday

International News via Reuters:

Euro zone companies are facing a slowing economy and increased competition from China as U.S. tariffs dent confidence and force rivals to seek new markets, a European Central Bank poll showed on Friday.

The ECB left interest rates unchanged on Thursday and offered a modestly upbeat assessment of the euro zone economy, raising doubts among investors about further policy easing even as U.S. tariff threats cloud the outlook.

But an ECB survey of 72 large companies operating in the euro area pointed to a slowdown in the manufacturing and services sectors, resulting in a more subdued outlook for employment and prices.

The ECB contacted the companies between June 23 and July 2.

“Contacts reported a slowdown in activity in recent months as tariffs, geopolitical tensions and the resulting uncertainty dented business and consumer confidence,” the ECB said. “The feedback from contacts was consistent with very modest growth in both the second and third quarters.”

The companies contacted by the ECB viewed U.S. tariffs – the extent of which is currently being negotiated – as a negative for growth and said competition from Chinese goods was playing an “increasing role”.

“The downward pressure on both activity and prices reflected reduced demand, in part caused by trade diversion from Asia (and China in particular) as exporters from the region sought alternatives to the U.S. market,” the ECB said.

This had mostly affected intermediate goods so far and had “little to no impact on final consumer prices,” but it was expected to broaden in the coming months and quarters.

“By contrast, contacts in the retail and consumer services sectors reported minimal, if any, impact on their activity or prices to date, and did not anticipate much impact in the near future,” the ECB added.

Wage growth was expected to slow from the 4.5% pace recorded last year, but by less than in the previous survey. Companies now forecast pay rising by 3.3% this year and by 2.8% in 2026.

Reporting by Francesco Canepa; Editing by Jamie Freed

The Belgian economist would now become Lagarde’s principal counsellor and the coordinator of the counsel to the ECB’s Executive Board

International News via Reuters:

The European Central Bank said on Friday it had picked Isabel Vansteenkiste as President Christine Lagarde’s new top adviser, replacing Roland Straub who would take up a new role at the ECB.

Vansteenkiste, currently director general for the ECB’s international and European relations, rose to prominence during the debt crisis of 2010-12, when she represented the central bank in the so called troika of institutions that bailed out Portugal, among other countries.

The Belgian economist would now become Lagarde’s principal counsellor and the coordinator of the counsel to the ECB’s Executive Board, a role that Straub had occupied since 2017.

The German economist was now set to become the deputy director general of the ECB’s market operations. Both appointments would become effective on 15 September, the ECB said.

Reporting By Francesco Canepa