AveXis marks opening of Longmont plant

LONGMONT — AveXis Inc. marked the completion of retrofitting its 692,000-square-foot drug-manufacturing campus in Longmont Tuesday morning, capping off a year of regulatory victories and intense criticism within the health-care industry.

AveXis’ drug production plant in Longmont. Courtesy Binswanger.

Before an assembled crowd of employees and public officials, AveXis president Dave Lennon said the company views the Longmont plant as the future for producing its flagship drug Zolgensma and gene therapies for other conditions in the company’s pipeline.

“Longmont will be on the leading edge of the industry transformation,” he said. “Longmont will be able to make Zolgensma for patients with SMA all over the world and will also support expansion of our pipeline impacting the lives of patients and families fighting rare genetic disorders.”

Zolgensma is a one-time intravenous injection that contains the effects of spinal muscular atrophy, a genetic disorder that prevents newborns from developing the strength needed to move body parts unassisted. Most patients die before they reach the age of 2.

Ciji Green, a Grand Junction resident whose daughter Maisie has SMA, also appeared at the event. Maisie’s story drew a wide following on social media last summer, which pressured the Greens’ health-insurance company to approve payment for her Zolgensma dose after an initial denial.

Plant manager Sally Dyer said the plant is due to begin making test batches of Zolgensma as part of a larger licensing process that is expected to span around 12 months. She estimates that the plant can make between 800 to 1,200 doses of the drug annually once at full capacity.

The plant currently has 347 employees, about 150 of whom are returning to the plant after drugmaker AstraZeneca PLC (NYSE: AZN) left the plant last March. Dyer expects the plant to employ about 400 people by year’s end.

Chris Lewis, a quality-control manager at the plant, said AveXis’ efforts to get the plant running again kept a large figure of highly-trained professionals from leaving the Front Range in search of pharmaceutical jobs elsewhere.

“Having that huge talent pool of immediately available employees was a big draw for us,” he said.

 

AveXis’ busy year

The year 2019 was among the most tumultuous years in AveXis’ history. The U.S. Food and Drug Administration approved Zolgensma for market in May at $2.1 million per dose, making it the world’s most-expensive drug per unit.

The FDA later said AveXis employees manipulated early animal-testing data and didn’t notify regulators until after it was approved, drawing criticism from members of the U.S. Senate. The drug’s approval was not revoked.

In October, federal regulators put a partial halt on clinical trials for Zolgensma for patients older than 2 due to safety concerns. Lennon said the company is cooperating with the FDA and hopes to hear back on restarting the trials this quarter.

Late last month, AveXis said it would give away up to 100 units of Zolgensma in a lottery during 2020 in outside of the U.S., where the drug has yet to receive regulatory approvals for sale abroad.

While the company said it developed the program with input from bioethicists, patient-access groups and other bioethics thinkers derided the program as a symptom of greater structural issues with the U.S. drug-pricing system.

Lennon disagreed with calling the giveaway a lottery, saying patients will be drawn randomly only if more than 100 families request the drug. He also said that 100-dose figure could increase in the future as the company adds more production capability.

“We know that there are more babies out there that we can possibly help with these hundred doses, and this is the fairest, most equitable way to actually allocate that,” he said.

While he declined to say if AveXis or Novartis are considering lowering the drug’s price as scale rises, he said the sticker price tag is “fair and equitable” when considering the cost of researching the drug and setting up production plants in Longmont, North Carolina and in the Chicago suburbs.

He also argued that private insurers are saving costs over time because they spend less in long-term hospitalizations and other treatments, but said individual state Medicaid plans are still proving a challenge in securing coverage for patients.

The only other FDA-approved SMA treatment is Spinraza, which costs $750,000 for the first year of treatment and $375,000 annually for the rest of the patient’s life.

“There are so many ways payers need to recognize that there is a tremendous long-term benefit, and they actually make their money back very quickly,” he said.

An earlier version of this story incorrectly stated Ciji and Maisie Green lived in Castle Rock. It has been corrected to Grand Junction.

LONGMONT — AveXis Inc. marked the completion of retrofitting its 692,000-square-foot drug-manufacturing campus in Longmont Tuesday morning, capping off a year of regulatory victories and intense criticism within the health-care industry.

AveXis’ drug production plant in Longmont. Courtesy Binswanger.

Before an assembled crowd of employees and public officials, AveXis president Dave Lennon said the company views the Longmont plant as the future for producing its flagship drug Zolgensma and gene therapies for other conditions in the company’s pipeline.

“Longmont will be on the leading edge of the industry transformation,” he said. “Longmont will be able to make Zolgensma for patients with SMA all over the world and will also support expansion of our pipeline impacting the lives of patients and families fighting rare genetic disorders.”

Zolgensma is a one-time intravenous injection that contains the effects of spinal muscular atrophy, a genetic disorder that prevents newborns from developing the strength needed to move body parts unassisted. Most patients die before they reach the age of 2.

Ciji Green, a Grand Junction resident whose daughter Maisie has SMA, also appeared at the event. Maisie’s story drew a wide following on social media last summer, which pressured the Greens’ health-insurance company to approve payment for her Zolgensma dose after an initial denial.

Plant manager Sally Dyer said the plant is due to begin making test batches of Zolgensma as part of a larger licensing process that is expected to span…