2025年孤儿药报告：孤儿药真的那么与众不同吗？

Are Orphans That Different? Are Orphans That Different? Orphans’ growth advantage over non-orphans is narrowing, and category leader Darzalex set toexceed $16 billion by 2030. Are orphans really a class apart? By Melanie Senior Are Orphans That Different? Orphans’ growth advantage over non-orphans is narrowing, and category leader Darzalex set to exceed$16 billion by 2030. Are orphans really a class apart? Worldwide Orphan Drug Sales & Share of Prescription Drug Market (2020-30) By 2030, orphan drugs will make up fifth of theforecast $1.6 trillion in worldwide prescriptiondrug sales – a share that has doubled over thelast decade. Orphans will continue to outpace theirmainstream counterparts for a few years yet: thecategory’s 2025-2030 forecast compoundannual growth rate (CAGR) tops 10%, versus7.5% for non-orphan prescription drugs. But orphans’ growth advantage looks set to slipto just 1% by the end of the decade, potentiallymarking the end of an extraordinary run. Orphans’ growth advantagelooks set to slip to just 1% by theend of the decade, potentiallymarking the end of anextraordinary run. Buoyed by considerable regulatory andexclusivity advantages, some orphan drugs nowresemble mainstream pharmaceuticalblockbusters, and dynamics within some nichemarkets mirror those across more prevalentdisease areas. With growth rates for orphan andnon-orphan categories also converging, thisbegs the question: should orphans still beconsidered a class apart? Top Drugs and Companies:Darzalex Dominates Top Drugs and Companies:Darzalex Dominates Sales of the biggest orphan, Johnson &Johnson’s multiple myeloma drug Darzalex(daratumumab), will exceed $16 billion by theend of the decade – exceeding the sales peakreached by BMS/Pfizer’s clot-buster Eliquis(apixaban). Darzalex secures J&J firmly atop thelist of biggest companies by 2030 orphan drugsales. Yet Darzalex may also fall within the crosshairsof the Inflation Reduction Act’s lower negotiatedprices for the costliest Medicare drugs, just asEliquis has done. J&J is seeking to protect it byarguing that its subcutaneous Fasproformulation is a new medicine, but it’s a greyarea. Meanwhile, other blood cancer mega-orphans, including AbbVie’s Imbruvica andAstraZeneca’s Calquence will also face Darzalex may also fall within thecrosshairs of the InflationReduction Act’s lower negotiatedprices for the costliest Medicaredrugs. IRA-negotiated pricing this decade. Calquence’sforecast sales of close to $5 billion neverthelesskeep it just about within the 2030 top ten best-selling orphans; its IRA price won’t kick in untilJanuary 2027. Top Orphans Mirror Mainstream Drugs Top Orphans Mirror Mainstream Drugs sales will reach close to $15 billion in 2030. & Johnson in 2017. PAH was virtually unheard ofbefore Actelion’s pioneer vasodilator Tracleer(bosentan) was launched in 2001, accompaniedby a vigorous disease awareness campaign. The blurring lines between niche and non-orphan drugs are also evident elsewhere in thetop ten roster aslifecycle management tacticsandexpanding awarenessof less-prevalentdiseases inflate orphan sales. The third-largest orphan drug by forecast 2030sales is Winrevair (sotatercept) for pulmonaryarterial hypertension (PAH). The condition -characterised by thickening of the arteriescarrying blood from the heart to the lungs –affects fewer than 50,000 US individuals. YetWinrevair is now a core driver of Merck’scardiovascular franchise. Obtained throughMerck’s $11.5 billion acquisition of Acceleron in2021, Winrevair helps place the pharma firmeighth in the top ten companies by 2030 orphandrug sales, and fuels a 2025-2030 orphan salesCAGR of over 30%, more than three times thatof any other top ten firm in the space. (Seecompany top ten and growth rates, table below). Today, like the rare blood cancers, PAH is a BigPharma battleground. Winrevair faces J&J’snewly approved Opsynvi tablet combiningActelion legacy drug Opsumit (macitentan) withUnited Therapeutics’ Adcirca (tadalafil), aformulation of Lilly’s erectile dysfunction drugCialis. Vertex’s cystic fibrosis therapy Alyftrek will bethe second-biggest orphan drug in 2030, withforecast sales of over $8 billion. Approved inDecember 2024, Alyftrek is a three-drug combotaken once-daily and thus more convenient than$10bn-a-year predecessor Trikafta (which,though declining, will still be the eleventh best-selling orphan in 2030). Alyftrek is Vertex’s fifthcystic fibrosis transmembrane conductanceregulator (CTFR) modulator, and the latest in asuite of incrementally improved CTFR genecorrectors and longer acting potentiators thathave extended the biotech’s dominance in adisease area affecting fewer than 100,000people worldwide. Vertex’s combined orphan Amsterdam-based Argenx, meanwhile, isreading Big Pharma’s life cycle managementplaybook with a rare autoimmune diseasesfranchise built around Vyvgart (efgartigimodalfa). Vyvgart – forecast to be the tenth best-selling orphan in 2030, with sales approac