Twenty-two years in and Alnylam Pharmaceuticals is still not raking in any profit. But that’s a testament to the company’s R&D focus, because it takes more than one or two successful drugs to make a lasting mark on the biopharma industry.
That biotech pluck keeps Alnylam reliant on its RNAi acumen and a strong R&D engine, but also product launches that stick the landing.
“We're still not profitable, so we have to be very thoughtful about how we optimize and how we justify costs,” said Tolga Tanguler, chief commercial officer and executive vice president at Alnylam.
The company’s latest win came in March with a U.S. approval for its drug Amvuttra to treat ATTR amyloidosis with cardiomyopathy. Leading the commercial launch, Tanguler is aiming for Amvuttra to become a first-line treatment due to the severity of the rare disease and the genetic medicine’s ability to knock the illness down more thoroughly than other treatments.
“In some disease categories, doctors like to wait for the more effective option later. Given that this is fast progressing, doctors want to be able to treat this as quickly as possible,” Tanguler said. “One of the challenges in this disease is, when you lose cardiac function, you don’t get it back — if you wait long enough, it’s too late.”
A smaller approach
For more than two decades, Alnylam has worked to bring the Nobel Prize-winning RNAi technology into the rare disease market as a mid-size biotech with stiff competition. Amvuttra’s main competition in the cardiomyopathy space is Pfizer’s tafamidis franchise, known as the brands Vyndaqel and Vyndamax.
“What this is doing for us is building the muscles that we’ve exercised well in the rare and specialty spaces, and building them into more prevalence."
Tolga Tanguler
Chief commercial officer, Alnylam Pharmaceuticals
Tanguler’s position is unique — once the head of North America rare disease at Pfizer, that franchise was once under his jurisdiction. He also led commercial efforts for well-known drugs like Viagra and Eliquis. But leading the commercial team at Alnylam is different, Tanguler said, in a way that is much better suited for the rare disease market.
“At Pfizer, we were working as a Big Pharma rare disease model, and I don’t think that really works because while large organizations are good at scaling up and driving large indications, they are not always in tune with a smaller, more captive audience like these rare diseases,” Tanguler said.
Tanguler sees the competition as a positive for Alnylam because of Pfizer’s ability to educate physicians and patients about the disease, which is considered to be widely underdiagnosed.
“Obviously, we don’t have the type of resources that a large company like Pfizer could provide, but I look at our science as building a series of skyscrapers with a strong, single foundation,” Tanguler said. “And as a smaller company, we have the ability to go deep into customer segments where you offer value-based solutions.”
Going bigger
Although Alnylam has focused on rare diseases, the company is striving to add more prevalent diseases like hypertension to the docket, partnering with pharma giant Roche in 2023 to co-develop and co-commercialize the RNAi candidate zilebesiran. And Alnylam also has an Alzheimer’s disease program in the works with Regeneron Pharmaceuticals.
“What this is doing for us is building the muscles that we’ve exercised well in the rare and specialty spaces, and building them into more prevalence,” Tanguler said.
One challenge Alnylam faces is addressing patient access issues for genetic medicines like RNAi. Pricing plays a role in that conversation, but Tanguler said Alnylam is suited for the task.
“If we’re going to price something, we’re going to have to price it with what we call value-based agreements — to pay for performance and base it on clinical outcomes,” Tanguler said.
With four products on the market since 2018, Alnylam pulled in $1.6 billion in 2024, enough to mark sizable growth from the previous year but still eclipsed by a $2.4 billion operating budget.
Tanguler said that points to the company’s devotion to R&D, punching above its weight in tackling the next big challenges in science.
“It’s a privilege — when I joined Alnylam, I said to myself, ‘This is the next big thing,’” Tanguler said. “Here we are, standing alone with amazing science and introducing these medicines in the most innovative way.”
