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Scientific breakthroughs in rare disease are coming at a faster clip as technology improves. But commercializing these drugs to a small population remains a challenge, and rare disease specialist Alexion has had to pave many new roads along the way.
Chief among those hurdles are finding the patients diagnosed with a certain condition and working with regulatory bodies to design a mechanism for approval. Alexion’s senior vice president of the U.S. business, Scott Weintraub, views these obstacles as an opportunity to be the first company that tackles specific unmet needs.
Now part of pharma giant AstraZeneca after a $39 billion buyout that closed in 2021, Alexion’s strategy requires even more foresight and communication between disparate departments of R&D and commercial than companies developing drugs for more widespread conditions, he said.
“In the Venn diagram between development and commercialization in rare diseases, the middle is pretty large, because you have to think about these things early on,” Weintraub said. “It’s going to be more than just the drug profile, which is obviously important, but can you find these patients?”
From locating patients to providing evidence to regulators, everything is a little bit harder when you’re on the cutting edge.
“If you’re the first, you have a lot more to do than just selling the drug,” Weintraub said. “A lot of times, you don’t have a path forward with the FDA at the time of your idea, so you have to convince them as to what’s important to these patients.”
Weintraub says that rare disease drug development needs to “start with the end in mind.”
Charting new waters
Strensiq is a drug that exemplifies those challenges, Weintraub said. The treatment for a rare bone condition called hypophosphatasia addresses a disease that’s typically difficult to diagnose in babies and children with fragile bones. The disease can also have a later onset, and it’s often misdiagnosed as fibromyalgia.
On the surface, what should be a market where Alexion has no competition is tough to penetrate because patients aren’t readily apparent — the company had to learn over time how many patients existed.
At the same time, the original formulation of Strensiq requires multiple injections a week for the rest of a patient’s life, a regimen that has evoked emotional pleas to cut down on the number of shots, Weintraub said. Now, Alexion is conducting a late-stage trial with fewer injections and a wider pool of patients.
“Patients in the trial have broken down in front of doctors, their life entirely consumed by getting their son these injections. That they’d be able to control his disease with a shot once every other week and not be reminded of it every day was really impactful for them,” Weintraub said.
Weintraub said these lessons from the patient community are especially important in rare disease.
“All of the challenges and opportunities in rare disease are difficult for development, and they’re difficult for commercialization — but at the same time, we would call that mission the secret sauce of Alexion,” Weintraub said. “If you’re in accounting or in a bench laboratory, you all know the mission.”
Following Soliris
The business of follow-on drugs is not new to the company. As Amgen was preparing to launch a biosimilar of Alexion’s paroxysmal nocturnal hemoglobinuria blockbuster drug Soliris in March next year, Alexion was racing the clock to deliver a new version called Ultomiris. Their mission was not only to get the drug approved, which happened in 2018, but to convince patients to switch.
During the AstraZeneca acquisition, the sustainability of the Soliris business was a key concern. And for rare disease patients, part of that solution was to reduce the injection burden from once every two weeks to only six times a year.
“A lot of those questions have gone away,” Weintraub said, pointing to Ultomiris’ lower dosing regimen as well as a 30% lower price compared to Soliris even though Ultomiris could have been sold at a higher price point. Alexion has also been exploring other indications for, gaining approval in 2022 for the rare neuromuscular disease generalized myasthenia gravis, or gMG, and most recently this year for neuromyelitis optica spectrum disorder.
“If you’re the first, you have a lot more to do than just selling the drug. A lot of times, you don’t have a path forward with the FDA at the time of your idea, so you have to convince them as to what’s important to these patients.”
Scott Weintraub
Senior vice president, U.S. business, Alexion
And with AstraZeneca on board, Alexion, which had previously operated in about 20 countries, has access to 70 markets worldwide.
In all of those markets, physician education is paramount to Alexion’s success, Weintraub said.
“A neurologist who has two gMG patients has a lot of questions about clinical trial data or real-world evidence, or the safety of X, Y and Z treatments,” Weintraub said. “If gMG was a paragraph in their medical books when they went through school, we can provide additional value to them over time.”
Ironically, the work Alexion does to pave the way in rare diseases also leads to further competition down the road — “because we created a pathway for drug approval, and once that mechanism is in place, other companies can at least get their drug approved and figure out their strategy.”
AstraZeneca’s promise
AstraZeneca CEO Pascal Soriot earlier this year promised 20 new medicines launched by 2030 and total revenue above $80 billion. As a $6 billion business unit, Weintraub said, Alexion’s leaders hope to have an outsized impact on achieving those goals.
“We’ll have at least a nice sized portion of the new molecular entities, and Alexion will punch above its weight to contribute to that,” Weintraub said, pointing out the company has 10 phase 3 programs in the works.
At AstraZeneca, Alexion now has access to a cutting-edge genetics portfolio that the pharma giant had been accumulating over time through biotech funding. Alexion also gained technology in the fields of diagnosis and patient tracking that make finding unmet rare disease needs easier than ever before.
“If you think about the diagnostic challenge, it takes an average of nearly five years and seven different specialists to even get the right diagnosis,” Weintraub said. “You can’t just go to market — you have to help with diagnosis and education around the globe, and understand the patient journey and what’s meaningful to them in terms of improvement.”
