4 Rare Disease Drugmakers With Strong Long-Term Growth Prospects

Rare diseases have moved steadily from being an overlooked corner of medicine to an important frontier for drug innovation. The FDA estimates that more than 7,000 rare diseases affect more than 30 million Americans, while most of these conditions still lack approved treatments. The agency defines a rare disease as one affecting fewer than 200,000 people in the United States, a threshold that has supported a specialized regulatory framework and incentives for orphan-drug development.

The scientific case is equally compelling. Many rare diseases are caused by genetic changes, making them suitable for targeted treatments such as RNA interference (RNAi), antisense oligonucleotides, enzyme replacement and gene therapy. Advances in molecular biology and genetic medicine are enabling drugmakers to target the root causes of these diseases instead of just managing symptoms. At the same time, the large collective patient population and high unmet need create significant commercial opportunities for therapies that can demonstrate meaningful clinical benefits.

Against this backdrop, Alnylam Pharmaceuticals ALNY, BioMarin Pharmaceutical BMRN, Ultragenyx Pharmaceutical RARE and BridgeBio Pharma BBIO stand out. While their platforms differ, all four have commercialized therapies targeting rare or genetically defined disorders and are advancing additional programs to extend their growth runways. Their combination of established product revenue, pipeline catalysts and exposure to diseases with substantial unmet needs makes these stocks worth watching for long-term growth opportunities. All four companies currently carry a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Alnylam Pharmaceuticals

Alnylam has built a leading RNAi franchise around genetically validated disease targets. Its commercial portfolio includes Amvuttra and Onpattro for hereditary transthyretin (ATTR) amyloidosis, Givlaari for acute hepatic porphyria and Oxlumo for primary hyperoxaluria type 1. Amvuttra is also approved for ATTR cardiomyopathy (ATTR-CM). In the first half of 2026, AMVUTTRA generated $1.90 billion in global net product revenue, up 137% year over year, while Givlaari and Oxlumo contributed $164.2 million and $103.4 million, respectively. Total product revenue increased 94% to $2.21 billion, driven primarily by higher demand for Amvuttra, particularly among U.S. ATTR-CM patients.

Year to date, ALNY shares have plunged 39.4% against the industry’s 8.7% growth.

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Looking ahead, Alnylam has several pipeline opportunities that could extend its growth beyond its current commercial portfolio. The most immediate opportunity is cemdisiran, which Alnylam is developing in partnership with Regeneron. The FDA is currently reviewing a new drug application (NDA) for cemdisiran monotherapy in generalized myasthenia gravis under the priority review pathway, with a decision expected in November 2026. The candidate is also under review in the EU for the same indication. Cemdisiran is also being evaluated in phase III studies for additional indications, including paroxysmal nocturnal hemoglobinuria and geographic atrophy.

Beyond cemdisiran, Alnylam’s other potential longer-term growth drivers include nucresiran, being developed in separate late-stage studies for hereditary ATTR polyneuropathy and ATTR-CM. Mivelsiran is being studied in separate phase II studies for cerebral amyloid angiopathy and Down syndrome-associated Alzheimer’s disease, while ALN-6400 is undergoing mid-stage development for hereditary hemorrhagic telangiectasia and von Willebrand disease. ALN-HTT02 is being evaluated in a phase Ib study for Huntington's disease.

BioMarin Pharmaceutical

BioMarin is a leading global rare-disease biotechnology company focused on developing medicines for genetically defined conditions, supported by a broad commercial portfolio. In the first half of 2026, Voxzogo, indicated for achondroplasia, generated $472.4 million, up 9% year over year, driven by higher sales volume from new patients. Naglazyme, for mucopolysaccharidosis type VI, generated $265.3 million, up 9%, while Palynziq, for phenylketonuria, brought in $224.7 million, up 13%, supported by higher patient starts, primarily in the United States. Galafold, approved for Fabry disease, contributed $105.7 million, and Pombiliti plus Opfolda, for Pompe disease, generated $30.3 million following BioMarin’s acquisition of Amicus in April 2026.

Year to date, BMRN stock has gained 9.8% compared with the industry’s 8.7% growth.

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Beyond its commercial portfolio, BioMarin has several opportunities to extend its rare-disease growth runway through label expansions, clinical-stage programs and business development. The FDA is currently reviewing BMRN’s application seeking full approval of Voxzogo in children with achondroplasia, with a Feb. 28, 2027, decision date. Additionally, it also submitted a supplemental NDA for Voxzogo to treat hypochondroplasia in July after phase III success. The company has also advanced the drug in clinical studies for two other short-stature pathway conditions — idiopathic short stature and Noonan Syndrome — in separate mid-stage studies.

BioMarin also has several potential growth drivers beyond Voxzogo. BMN 333, a long-acting CNP, entered a registration-enabling phase II/III study for achondroplasia in April 2026. BioMarin also acquired U.S. rights to BMN 820, a potential first-in-class oral CCR2 inhibitor for focal segmental glomerulosclerosis. Last month, BMRN agreed to acquire ALE1, an oral phase I/IIa candidate for hypophosphatasia, for $275 million upfront plus potential milestone payments, from Alesta Therapeutics.  Together with the expanding Voxzogo franchise and the newly added Amicus assets, these programs provide avenues for extending growth well beyond the company's current products.

Ultragenyx Pharmaceutical

Ultragenyx is perhaps the clearest pure-play rare-disease name in the group, focused on developing and commercializing therapies for serious rare and ultra-rare genetic diseases. Its four commercial products are Crysvita for X-linked hypophosphatemia and tumor-induced osteomalacia, Dojolvi for long-chain fatty-acid oxidation disorders, Evkeeza for homozygous familial hypercholesterolemia and Mepsevii for mucopolysaccharidosis type VII. RARE commercializes Evkeeza outside the United States under its partnership with Regeneron. In the first half of 2026, total revenues increased 14% to $350 million. Growth was primarily driven by higher Crysvita sales, particularly in Latin America, due to an increase in patients and the favorable timing of shipments, as well as continued international expansion of Evkeeza.

Year to date, RARE shares have gained 9.6% compared with the industry’s 8.7% growth.

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Ultragenyx has since added another commercial asset. Last month, the FDA granted accelerated approval to Genglycos (DTX401) for adults and children at least eight years old with glycogen storage disease type Ia, making it the company's fifth FDA-approved therapy. Meanwhile, an NDA for UX111 to treat Sanfilippo syndrome type A is currently under FDA priority review and has a Sept. 19, 2026, decision date.

RARE has several late-stage pipeline candidates. GTX-102 is in phase III development for Angelman syndrome, with pivotal data expected in September or October 2026. UX701, an investigational AAV9 gene therapy, is being evaluated in a phase I/II/III Cyprus2+ study to treat Wilson disease and expects to share top-line data in the fourth quarter. DTX301 is also undergoing late-stage development for ornithine transcarbamylase deficiency.

BridgeBio Pharma

BridgeBio is a commercial-stage biopharmaceutical company focused on medicines for genetic diseases. Attruby (acoramidis) is its sole major commercial product in the United States, marketed as Beyonttra internationally, approved for ATTR-CM. In the first half of 2026, product revenues surged 272% year over year to $403 million, reflecting continued commercial expansion and strong patient demand for Attruby. The company is also evaluating the potential kidney-protective benefits of acoramidis, while a recent government agreement is expected to broaden access to Attruby for Medicaid patients.

Year to date, BBIO shares have gained 0.1% compared with the industry’s 6.3% growth.

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Looking ahead, BridgeBio has three late-stage candidates that could broaden its rare-disease portfolio. BBP-418, an oral therapy for limb-girdle muscular dystrophy type 2I/R9, is currently undergoing FDA priority review, with a Nov. 27, 2026, decision date. An NDA for encaleret, a potential targeted treatment for autosomal dominant hypocalcemia type 1, is also currently under FDA review with a decision expected on May 8, 2027. Additionally, BridgeBio has submitted an NDA seeking FDA approval of low-dose infigratinib for achondroplasia. BBIO is also evaluating these candidates for additional indications, with encaleret being evaluated in a phase III study for chronic hypoparathyroidism and infigratinib in a phase II/III study for hypochondroplasia.

BridgeBio is also expanding its longer-term opportunity around acoramidis. The company recently dosed the first patient in ASCEND-ATTR, a phase IIIb/IV study evaluating the long-term effects of acoramidis on cardiac structure, function and amyloid burden in ATTR-CM.

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