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Several factors come into play to dull orphan drugs' shine such as the “blazing return of big drugs for big diseases,” according to Evaluate. The orphan drug sector has long been clearing growth in the larger pharmaceutical market with booming sales. But, thanks to blockbuster meds across wider disease areas and other challenges, the lucrative market may soon slow down for the first time in years. According to Evaluate’s 2024 report, “Orphan Drugs are Losing Their Sparkle,” the category is set to pick up $185 billion this year and some $270 billion by 2028. Yet, sales growth is notably shrinking. The meds averaged an almost 11% sales increase in the decade ending in 2023 but will "barely hit double digits" through 2030.  Several factors come into play to dull orphan drugs' shine, such as the “blazing return of big drugs for big diseases,” according to Evaluate. While the obesity market is currently the hottest ticket, blockbusters across disease areas such as neuroscience, immunology and oncology also stand to overtake orphan drug’s slice of the pie. Plus, there are pricing pressures stemming from the high price tag on orphan drugs. Payers are “more discerning” as of late and aren’t as keen to cover the steep prices, favoring more widespread disease areas instead, according to Evaluate.  Still, the sales slowdown is “relative,” head of thought leadership at Evaluate Daniel Chancellor said in a press release. “As the overall prescription drug market continues to grow, so will that for rare diseases.” 2028's top orphan drugs   According to the report, the top 10 most valuable orphan drugs will together collect more than $57 billion in 2028. That top 10 list has had a bit of a shakeup since Evaluate’s last ranking in 2023, largely due to increasing competition and the Inflation Reduction Act. Johnson & Johnson’s multiple myeloma blockbuster Darzalex, Vertex’s cystic fibrosis med Trikafta and Roche’s hemophilia A therapy Hemlibra all kept their top three slots on Evaluate’s list of the 10 top sellers by 2028 in worldwide sales with predicted respective revenues of $17 billion, $8.7 billion and $6.2 billion. However, AbbVie’s popular cancer med Imbruvica has fallen out of the top 10 thanks to Beigene’s rival BTK inhibitor Brukinsa and AstraZeneca’s Calquence. While Calquence is slated for $4 billion in 2028 sales and Brukinsa should pick up $3.8 billion, AZ’s offering fell from fifth place to seventh over the past year. Argenx’s Vyvgart also slipped, dropping to 14th place with a forecast $3 billion in 2028. The generalized myasthenia gravis drug is challenged by growing competition such as UCB’s two new treatment options, Zilbrysq and Rystiggo, plus upcoming threats from Johnson & Johnson’s nipocalimab. Despite label expansions last year, Roche’s spinal muscular atrophy therapy Evrysdi had a similar story with competition from Biogen's Spinraza and Novartis' Zolgensma. Meanwhile, new entrants to the top 10 list included Incyte and Novartis’ JAK inhibitor Jakafi, which placed sixth with a 2028 sales expectation of $4.2 billion, and J&J’s Carvykti. The latter is the only CAR-T therapy to appear on the top 10 list with $4.4 billion in predicted 2028 sales. As for the wider 2028 picture, Amgen swiped AbbVie’s spot on the list of the top 10 companies with largest orphan drug sales. That can be attributed to Amgen’s $27.8 billion acquisition of rare disease drugmaker Horizon Therapeutics, which brings orphan drugs Tepezza, Krystexxa and Uplizna into the fold. The top spots in that ranking go to J&J, Roche and AZ, with J&J taking No. 1 in a landslide. The company is expected to bring in over $30 billion in 2028 orphan drug sales, while Roche at No. 2 looks to take home $16.8 billion.

Biogen said Wednesday that sales of its Eisai-partnered Alzheimer's disease treatment Leqembi (lecanemab) gained traction in the first quarter, reaching $19 million and nearly tripling the sales it generated in the previous three-month period.According to Biogen, a "significant increase" in new patient starts was seen in March, with CEO Christopher Viehbacher noting that "momentum [is] building at a steady pace" for the product. "As the launch progresses and infrastructure develops, we continue to believe in the potential longer-term commercial opportunity in Alzheimer's disease," he added.However, the performance was still far below the $30 million Jefferies analysts had forecast, although other analysts had set a more modest $11-million benchmark. Still, investors were pleased, bumping up company shares by nearly 7% before the bell.Pace of patient startsBiogen said the number of patients on the anti-amyloid antibody had increased by nearly 2.5-fold from roughly 2000 patients at the end of 2023. Eisai had previously aimed to treat 10,000 patients by March, but earlier this year indicated that it would likely miss that target. For more, see Physician Views In-Depth: Neurologists provide five key insights into Leqembi's laggardly launch.While the latest numbers are encouraging, Jefferies analyst Michael Yee said the growth in patient starts needs to pick up steam from here.Leqembi's uptake has been hindered by requirements such as extra diagnostic testing, twice-monthly infusions and regular brain scans. Viehbacher said challenges include the "amount of effort it takes to actually be able to initiate even the first patient…I do think we're now on a positive track. I think we're seeing momentum," although he does not expect the launch to be linear.Bright spot with new launchesBiogen came into 2024 riding on the momentum of three other key product launches last year. Aside from Leqembi, the company also secured regulatory nods for Zurzuvae, a drug for post-partum depression (PPD) co-developed with Sage Therapeutics, and for Qalsody (tofersen), which is used to treat an ultra-rare genetic form of ALS. It also expanded in the rare disease space with a drug for Friedreich's ataxia, marketed as Skyclarys (omaveloxolone), when it acquired Reata Pharmaceuticals for $7.3 billion.Zurzuvae notched approximately $12 million in quarterly revenue, whereas analysts had expected just $5 million for the drug, which missed out on the larger opportunity in major depressive disorder when the FDA approved it only for the PPD indication. See Spotlight On: Zurzuvae approval a real downer for Sage, Biogen.Meanwhile, Skyclarys brought in $78 million, which edged passed consensus by about $6 million, while Qalsody generated $4.6 million in sales.Older drugs continue to struggleHowever, Biogen's mainstay MS drugs and spinal muscular atrophy treatment Spinraza continue to grapple with increased competition. Sales of its once-blockbuster MS drug Tecfidera came in at $254.3 million, although that was still about $18 million higher than consensus, while the MS franchise saw sales dip 4% to $1.1 billion during the quarter. Spinraza had sales of $341.3 million, falling below estimates.Overall sales for the first quarter were down 7% at $2.3 billion, which was in line with consensus, while net income reached $393.4 million, up from $387.9 million for the same period last year. Viehbacher indicated that the company's cost-cutting measures are also taking hold and helped offset competition for its older drugs. Last July, Biogen said it would eliminate 1000 jobs, or more than 11% of its workforce, in a bid to rein in operating expenses by an additional $700 million by 2025.Biogen on Wednesday also reaffirmed its outlook for adjusted earnings this year of $15 to $16 a share. The CEO said Biogen has no upcoming plans for major acquisitions, but is instead interested in licensing drugs and collaborating with other companies to expand its portfolio beyond neuroscience and into rare diseases and immunology.