Sarepta Stock is located on the FDA genes box star-news.press/wp

shares Sarepta treatments More than 30 % decreased on Friday, when the future of adopted genetic therapy appeared.

The Food and Drug Administration will ask the company to stop all treatment shipments voluntarily, a person familiar with the CNBC command.

Sarpta told CNBC that she had not heard from the Food and Drug Administration.

Separately, Marti Macari, Commissioner of FDA Tell Bloomberg News The agency studies whether the company’s genetic therapy should remain on the market.

The FDA (FDA) is investigating the death of two patients associated with Elevidys, which represent more than half of the net product revenue in Sarepta. The company also reported a third death linked to a separate experimental genetic treatment.

Elevidys are drowned in controversy even before approval. Genetic therapy has not yet been proven that it can benefit people with Duchenne muscle atrophy, a condition that erodes muscle function over time.

People with the disease eventually lose the ability to walk, and most of them die by the early twenties, which means that there is a great need that is not necessary for treatment. The Food and Drug Administration (FDA) was originally granted a conditional approval of patients only between the ages of 4 and 5, the group that witnessed the greatest benefit in clinical trials.

The following year, the agency granted treatment full approval for patients 4 and above who can still walk and accelerate patients 4 and above who can no longer walk. The last decision was particularly controversial because there was less evidence that Elevidys could help people who have already made their disease a lot.

In addition, Elevidys failed to achieve its goal in experimenting with the third stage, although the company argued that the drug showed a promise of other standards in the study. Then the head of the Food and Drug Administration Center for the evaluation and research of biology Peter Marx agreed to evaluate Salabta FDA staff (FDA) has spent the expansion of the approval of Elevidys.

Earlier this year, Sarpta revealed that two teenage boys had passed away due to the failure of the liver after receiving it. Then this week, it was reports that another person had died during the trial of the first stage that was investigated in one of the treatments of genes that were sacrificed for a different disease.

The two the two treatment differ, although they share the same delivery method, which increases safety concerns about Elevidys. BMO Kostas Biliouris analyst said that safety risk from Elevidys is especially important given the uninterrupted benefit.

For example, the genetic therapy of Novartis also caused spinal muscles in the liver toxicity and death, but the benefit of this treatment is clear.

“For this reason, the deaths here are a lot against Zolgansma, for example.”

Zolgensma is just one drug from many for a large company like Novartis. For Sarepta, Elevidys is everything.

Executive officials this week have tried to reassure investors that even if they could treat patients who can still walk, as deaths have not been reported, treatment should bring at least $ 500 million annually. SAREPTA last month stopped recharged Elevidys to patients who can no longer walk while exploring a safer way to manage treatment.

Peliores said that the highest concern of investors at this stage is whether the Food and Drug Administration is withdrawing the drug. The shares of the company now decreased more than 87 % this year.

“If the food and drug administration withdraws Elevidys from the market, it is easy,” he says.

“It is surprising” that other patients will not have a treatment option to wrap if the shipments of the heights are stopped.

Her son, Charlie, with Elevidys in 2022 as part of the Sarepta experience late in the stage and noticed improvements in six to 12 months, including increased endurance and the most liquid suggestions. The drug also reduced the symptoms of the condition called Gowers, which causes the difficulty of children when waking up from sitting or lying.

She said that her son was “completely stable” for three years of his dose. Handt said she was familiar with the dangers of the liver toxicity before Charlie was recorded in the trial.

“We do not have the luxury of not risking,” said Handt. “There are families that dealt with this disease before that would have had any option, even if there were risks.”

“Each family must have the option to take this jump with this medicine and may see the benefits,” she added.