Rare Disease

Shionogi & Co., Ltd. announced that its Board of Directors approved an agreement to acquire IntraBio Inc., a biopharmaceutical company developing and commercializing therapies for neurodegenerative diseases, for an upfront consideration of USD 2.0 billion payable to IntraBio shareholders. Under the agreement signed on October 5, 2026, IntraBio would become a wholly owned subsidiary of New Jersey-based Shionogi Inc., with the transaction scheduled to close between November 2026 and December 2026, subject to competition-law waiting periods and other customary conditions. The deal would add AQNEURSA (levacetylleucine) to Shionogi's rare disease portfolio; the drug was approved by the FDA in September 2024 for neurological manifestations of Niemann-Pick disease type C and by the European Medicines Agency in January 2026, and on September 18, 2026 it became the first and only FDA-approved treatment for Ataxia in patients with Ataxia-Telangiectasia, for which it is also under EMA review. Shionogi said the acquisition builds on the rare disease foundation it established through its April 2026 acquisition of global rights to edaravone, known as RADICAVA in the U.S. and RADICUT in Japan, and would strengthen its pipeline across Pompe disease, Fragile X syndrome, Jordan's syndrome and early-stage rare neurodegenerative programs. The impact on Shionogi's consolidated financial results for the fiscal year ending March 2027 is currently under review.

CSL and Alentis Therapeutics have entered an exclusive global partnership to co-develop and co-promote lixudebart for rare kidney, liver, and other diseases. Under the agreement, CSL will arrange an initial payment of $355M to Alentis, which is also eligible for up to $1.2B in commercial milestone payments, bringing the total deal value to $1.55B. The companies will share global profits 55% to CSL and 45% to Alentis once the drug is commercialized. CSL will fund Phase 2 and planned Phase 3 studies of lixudebart in AAV-RPGN while advancing Phase 2 programs in FSGS and PSC. Lixudebart is currently being evaluated in the Phase 2 RENAL trial for AAV-RPGN, a rare autoimmune disease that can cause rapid kidney function loss and irreversible kidney damage.

AnnJi Pharmaceutical announced it is proceeding with the U.S. portion of its pivotal Phase 3 ROMA-KD trial of AJ201, also known as rosolutamide, in patients with spinal and bulbar muscular atrophy, or SBMA, also called Kennedy's disease. The company said it submitted the Phase 3 protocol to the U.S. FDA under its active Investigational New Drug application and will now activate U.S. sites for the global trial. The ROMA-KD study is a global, multicenter, randomized, double-blind, placebo-controlled trial expected to enroll approximately 200 ambulatory patients with symptomatic SBMA worldwide, with the United States as a key region, and is intended to support potential global regulatory submissions. AJ201, an investigational oral small molecule and a potential first-in-class treatment for SBMA, has received Fast Track Designation from the U.S. FDA and Orphan Drug Designation in both the United States and the European Union. AnnJi said the Phase 3 program builds on encouraging results from its completed Phase 2 study announced in May 2025, and the company also noted its SBMA Patient and Care Partner Advisory Council, first announced in collaboration with the Kennedy's Disease Association at the 2026 KDA International Patient and Scientific Conference.

Wells Fargo initiated coverage of Design Therapeutics with an overweight rating, citing the company's Friedreich ataxia candidate DT-216P2 as having potentially best-in-disease functional improvement based on results from the RESTORE-FA study released in May. The bank set a $26 price target, implying roughly 112% upside based on the October 1 close. Analyst TianQi Hang wrote that the May update showed pharmacokinetics look good, and that blood-FXN protein, muscle-mRNA data, plus an early mFARS signal further de-risk the platform. Hang estimates that the blood FXN protein increase seen after 6 weeks can translate to at least a 2-point mFARS change, and said that if the drug kinetics sustain for 12 weeks, which he believes they will, DT-216P2 could deliver best-in-disease functional benefits. He assigns DT-216P2 a 60% probability of success, with peak sales of approximately $600M in the US and approximately $900M outside it. If approved, DT-216P2 would compete against Biogen's Skyclarys, also known as omaveloxolone, and Hang sees it gaining a peak share of the FA treatment market of 30% in the US and 20% ex-US.

The US FDA has approved Bristol Myers Squibb's Camzyos, also known as mavacamten, for children with symptomatic obstructive hypertrophic cardiomyopathy, a rare genetic heart condition that can cause shortness of breath, fatigue, and abnormal heart rhythm. The approval was based on results of the phase 3 double-blind, placebo-controlled SCOUT-HCM trial, which enrolled children 12 to 18 years old with symptomatic oHCM; patients in the Camzyos arm had a significantly reduced Valsalva LVOT gradient, a measure of pressure in the left ventricular outflow tract while bearing down, compared to placebo. Camzyos carries a boxed warning for heart failure risk and is restricted through a Risk Evaluation and Mitigation Strategy program. The treatment was approved for adults in 2022.

argenx SE announced it will present new clinical, long-term and real-world data across its neuromuscular portfolio at the 2026 American Association of Neuromuscular & Electrodiagnostic Medicine Annual Meeting and the Myasthenia Gravis Foundation of America Scientific Session in Orlando, Florida, from September 29 to October 2, 2026. In myasthenia gravis, Phase 3 ADAPT OCULUS data showed ocular MG improvements deepened with additional VYVGART cycles, with mean MGII ocular scores improving from -4.5 to -6.8 points in AChR-Ab-positive patients and from -2.7 to -4.5 points in triple-seronegative patients, while one-year ADAPT SERON results showed mean MG-ADL improvements of approximately 5 points maintained through Week 52 in anti-AChR antibody-negative generalized MG. A real-world analysis of more than 1,100 U.S. MG patients found patients treated within a year of diagnosis had a mean MG-ADL reduction of 4.7 points over the first three months versus 3.5 points for those treated more than three years after diagnosis, with 50% reaching minimal symptom expression. In CIDP, an interim analysis of ADHERE and ADHERE+ showed approximately 40% of responding participants reached an INCAT score of 0 or 1 with follow-up beyond five years, a post hoc analysis found VYVGART Hytrulo reduced the relative risk of grip strength deterioration by 71.5%, and a Phase 4 switch study showed 87% of patients remained on VYVGART Hytrulo through 12 weeks after a one-week direct transition from IVIg. The company also reported sustained clinical benefit and consistent safety for empasiprubart in multifocal motor neuropathy from the Phase 2 ARDA+ study, and in-clinic and real-world walking improvements for adimanebart in DOK7-congenital myasthenic syndrome from a Phase 1b study.

Aptadir Therapeutics has closed a EUR 40M Seed round to advance a novel class of RNA inhibitor-based therapeutics for intractable genetic conditions. The round was led by 4BIO Capital, with follow-on participation from the company's original pre-seed investor EXTEND, Italy's National Technology Transfer Hub launched by CDP Venture Capital SGR and jointly funded by Angelini Ventures and Evotec SE. Additional support came from CDP Venture Capital through the Digital Transition Fund, Indaco Venture Partners, XGEN Venture, CE-Ventures, Angelini Ventures through a direct investment, Kerna Ventures, Italian Angels for Biotech, and Club degli Investitori. The funds will advance the company's pipeline of disease-modifying investigational RNA therapeutics, including its lead candidate CAP1-FMR1 for Fragile X Syndrome, based on a new class of RNAs called DNMTs Interacting RNAs that block aberrant DNA methylation at a single gene level to reactivate silenced gene expression. The science originates from the Beth Israel Deaconess Medical Center, the Italian Research National Council, and the Cancer Science Institute of Singapore.

Truist analyst Danielle Brill identified a significant positive readthrough for Maze Therapeutics following positive Phase 2 topline data from Vertex Pharmaceuticals, validating APOL1 inhibition in classic APOL1-mediated kidney disease and supporting an addressable population of over 100,000 patients. Truist reiterated a Buy rating and $64 target on Maze, noting the market is large enough to support blockbuster potential for Maze's MZE829 even if efficacy merely matches Vertex's inaxaplin. Maze reported $494.9 million in cash, cash equivalents, and marketable securities in Q2 2026, funding its Phase 2 HORIZON trial of MZE829 with cohort updates expected in late 2026 or early 2027 and a pivotal trial planned for H1 2027. Vertex, which posted $3.33 billion in Q2 2026 revenue, up 12% year-over-year, and holds $13.6 billion in cash and marketable securities, raised its full-year 2026 revenue guidance to $13.1–$13.2 billion. Maze faces late-mover risk behind inaxaplin and reported a Q2 2026 net loss of $44.7 million, while Vertex's R&D and SG&A expenses reached $1.6 billion in Q2 2026.

Biogen's growth portfolio generated $1.06 billion in second-quarter 2026 revenues, up 24% year over year and 25% sequentially, surpassing the company's legacy MS portfolio, which brought in $767 million. Even excluding newly acquired Syfovre and Empaveli, growth-product revenues reached $933 million, up 9% year over year and 10% quarter over quarter, still above the legacy MS portfolio. Within the growth portfolio, Skyclarys revenues rose 29% to $168 million, Zurzuvae increased 53% to $71 million, Spinraza grew 2% to $402 million, and Vumerity fell 7.4% to $196.5 million, while Syfovre and Empaveli contributed $97.4 million and $30.4 million respectively. Alzheimer's collaboration revenues from Eisai for Leqembi rose 16% to $63.7 million, and the companies expect blood-based diagnostics and the launch of Leqembi Iqlik to drive further growth from 2027 onward. Biogen expects the growth portfolio to maintain its lead in the second half of 2026, with Syfovre and Empaveli contributing more than in the second quarter since revenue recognition began only in mid-May.

Oryzon Genomics, S.A. announced that the European Medicines Agency has authorized its Clinical Trial Application to initiate a Phase II study of vafidemstat for the treatment of Phelan-McDermid Syndrome. The study, named HOPE-2, is a single-center, single-arm, open-label Phase IIa trial that will enroll 12 adult patients with PMS, a severely disabling genetic disorder related to autism with a U.S. prevalence estimated at approximately 1 in 7,300 people. The primary objective is to evaluate the safety and tolerability of vafidemstat, with secondary objectives assessing its effect on anger and aggression and overall disease efficacy; vafidemstat will be administered for 12 weeks, after which the investigator will assess whether participants may continue treatment through week 24 based on clinical benefit. The study will be conducted in Spain as part of Oryzon's VANDAM project, which is part of the Med4Cure Important Project of Common European Interest on Health and has received funding from the Spanish Ministry of Science, Innovation and Universities and the Centre for the Development of Industrial Technology and Innovation under the Recovery, Transformation and Resilience Plan funded by the European Union – NextGenerationEU. Oryzon will collaborate with the Spanish Phelan-McDermid Syndrome Association to support identification of potential participants.

The United States Food and Drug Administration granted platform technology designation to Precigen's AdenoVerse immunotherapeutic platform, which underpins the recently approved PAPZIMEOS therapy. The designation and the earlier PAPZIMEOS approval have coincided with a sharp shift in market sentiment toward Precigen, with the share price delivering an 81.07% year-to-date return and a three-year total shareholder return exceeding 4x, while the more recent 90-day share price return of 32.25% indicates momentum has been building. The stock last closed at $7.75, and the most followed analyst view puts fair value at $11, framing the rally as only a partial catch up. On that view, bearish analysts expect earnings to reach $253.7 million, or $0.68 per share, by about August 2029, up from $336.7 million of losses today, while more bullish analysts expect earnings as high as $363.7 million. On a sales-based measure, the stock trades at 32.4x sales against a US Biotechs group average closer to 12.1x and a fair ratio of 9.5x, a gap that points to valuation risk if sentiment or forecasts change.

The US FDA on Friday approved Mirum Pharmaceuticals and Incyte's Atebrioz, also known as zilurgisertib, as a treatment for the rare bone condition fibrodysplasia ossificans progressiva, or FOP. The oral activin receptor-like kinase 2 inhibitor is designed to reduce the volume of total new heterotopic ossification in adults and children 12 years and older. Approval was based on results from a cohort of the PROGRESS study that showed at week 24, mean total new heterotopic ossification lesion volume decreased by 3.2 cm³ in patients receiving Atebrioz compared with an increase of 24.6 cm³ in those on placebo, with benefits maintained through week 48 of the open-label extension. FOP is an ultra-rare, progressive genetic disease impacting approximately 300 people in the US and approximately 900 worldwide, characterized by heterotopic ossification in which bone forms in muscles, tendons, ligaments, and other soft tissues.

Sanofi's new and acquired drugs are emerging as a broadening growth base beyond its flagship immunology medicine Dupixent, with sales of those newer products rising 48.3% at constant exchange rates to €1.3 billion in the second quarter, led by Altuviiio, Ayvakit and Sarclisa. Dupixent, marketed in partnership with Regeneron, remains the company's primary growth engine and accounted for roughly 42% of Sanofi's first-half 2026 revenues. Altuviiio achieved blockbuster sales in 2025, while Ayvakit, added through the Blueprint Medicines acquisition, is expected to become the next blockbuster drug in 2026, and Beyfortus, developed with AstraZeneca, reached blockbuster sales in its first full year in 2024. Sanofi now expects its Pharma launches to generate approximately €10 billion of annual sales by 2030, compared with about €25 billion for Dupixent, as it positions the newer medicines to gradually reduce reliance on its top-selling drug. The Zacks Consensus Estimate for 2026 earnings has risen from $4.96 per share to $5.07 per share over the past 60 days, while 2027 estimates climbed from $5.18 to $5.29 per share, and Sanofi stock has fallen 15.5% year to date against an 11.7% gain for the industry.

Prime Medicine shares jumped 8% Friday after the FDA cleared its IND application for PM647, an experimental gene-editing therapy for Alpha-1 Antitrypsin Deficiency. The clearance allows Prime Medicine to begin a Phase 1/2 trial evaluating PM647 in adults with the inherited disorder, with initial clinical data expected in 2027. PM647 is designed to fix the genetic mutation that causes Alpha-1 Antitrypsin Deficiency, and Prime Medicine said the treatment could restore the normal protein and potentially help treat both the lung and liver problems caused by the disease.

Vertex Pharmaceuticals is building a differentiated kidney disease franchise to diversify beyond its dominant cystic fibrosis business, with povetacicept and inaxaplin seen as significant commercial opportunities. Povetacicept, added through the approximately $4.9 billion acquisition of Alpine Immune Sciences in 2024, targets the BAFF and APRIL cytokines, and the FDA accepted a regulatory filing for its use in IgA nephropathy in June 2026, with a final decision expected on Nov. 30, 2026. If approved, povetacicept would become Vertex's first marketed nephrology product, addressing an estimated 330,000 people with IgAN in the United States and Europe and more than 1.5 million diagnosed patients globally, with analysts forecasting blockbuster sales and peak annual revenues in the multi-billion-dollar range. Inaxaplin is being developed in the phase II/III AMPLITUDE study in primary APOL1-mediated kidney disease, with enrollment complete and interim data expected in early 2027, while data from the phase II AMPLIFIED study announced earlier this week showed a 42.7% reduction in urine albumin to creatinine ratio at week 13 in patients with AMKD and modest proteinuria, and a 17.3% reduction in those with AMKD and type II diabetes. The IgA nephropathy space has grown more competitive, with the FDA already approving Otsuka's Voyxact, Vera Therapeutics' Trutakna and Novartis' Vanrafia.

Otsuka Pharmaceutical and U.S.-based Ionis Pharmaceuticals announced on the 22nd that their jointly developed treatment for hereditary amyotrophic lateral sclerosis, ulefnersen, met its primary goal in a late-stage clinical trial. In patients with FUS mutation ALS, a rare inherited form of ALS that damages the nerve cells controlling movement, ulefnersen improved function and extended survival compared with the placebo group. The drug reduced markers of nerve cell damage and slowed disease progression, and most side effects were mild or moderate, indicating a favorable safety profile. No approved treatment currently targets the genetic cause of FUS-ALS, and the two companies plan to discuss the results with the U.S. Food and Drug Administration and global health authorities as they explore a path toward accelerated approval. Separately, Otsuka Pharmaceutical has launched a global early access program for FUS-ALS patients unable to participate in the trial, allowing physicians to request access to ulefnersen before approval.

Arcturus Therapeutics said interim phase 2 results for ARCT-810 showed the candidate reduced and/or maintained first morning fasting ammonia within the normal range in patients with ornithine transcarbamylase deficiency, and also reduced glutamine, with some individuals reaching the normal range. Based on those results and a June meeting with the US FDA, the company aims to begin dosing of ARCT-2601 close to the end of the year in participants 12 years and older under an amended phase 2 protocol that integrates ARCT-2601 into the current ARCT-810 phase 2 study. Both ARCT-810 and ARCT-2601 are mRNA therapeutics for OTC deficiency, but ARCT-810 uses the LUNAR platform while ARCT-2601 uses the next-generation LUNAR 2.0 platform, which Arcturus says produces greater than 30-fold higher protein expression and could allow for lower or less frequent dosing. OTC deficiency is a rare genetic disorder in which the body stops breaking down and removing nitrogen, leading to dangerous ammonia levels in the blood. Arcturus is also acquiring AI discovery company myNeo, with which it has worked since 2024, and the deal is expected to close in October.

Roche Holding partner Ionis reported that sefaxersen met the primary endpoint in the Phase 3 IMAgINATION trial for IgA nephropathy, delivering a statistically significant reduction in proteinuria compared with placebo. The clinical win comes on top of a strong run for Roche Holding, with the share price up 8.32% over 90 days and 11.58% year to date, and a 1-year total shareholder return of 44.02%. Roche Holding now trades at CHF363.20, only about 3% below the average analyst target, yet screens at a roughly 59% discount to an intrinsic value estimate, while the most followed narrative fair value of CHF353.34 pegs the stock as 2.8% overvalued. The SWS DCF model points the opposite way, implying a future cash flow value of CHF893.70. The story could still change quickly if key Phase 3 programs disappoint or if biosimilar pressure on older blockbusters accelerates faster than expected.

Ionis Pharmaceuticals announced positive late-stage results from two partnered phase III programs. The FUSION study of ulefnersen in amyotrophic lateral sclerosis caused by mutations in the fused in sarcoma gene met its primary endpoint assessing functional impairment and survival at 72 weeks, with Ionis reporting the results as statistically significant without disclosing supporting numbers; Otsuka Pharmaceutical, which holds worldwide commercialization rights licensed in 2024, plans to discuss the data with the FDA and other global health authorities regarding potential expedited regulatory submission pathways. Separately, the IMAgINATION study of sefaxersen in adults with primary immunoglobulin A nephropathy, conducted by Ionis' partner Roche, met its primary endpoint in a prespecified interim analysis, showing statistically significant and clinically meaningful reductions in proteinuria after 37 weeks, and will continue in a blinded manner to evaluate kidney function over two years with estimated glomerular filtration rate at week 105 as the longer-term measure. Roche licensed sefaxersen from Ionis in 2022 and is responsible for the phase III study and future global development, regulatory and commercialization activities, while Ionis is eligible for milestone payments and tiered royalties on net sales of both drugs. The two wins follow back-to-back cardiovascular setbacks for Ionis, including the phase III CARDIO-TTRansform failure of Wainua with AstraZeneca in July and the phase III Lp(a)HORIZON miss for pelacarsen with Novartis, and come after the FDA approval of Zanvastro for Alexander disease earlier this month. Year to date, Ionis shares have lost 42% compared with the industry's 2% decline.

Vertex Pharmaceuticals announced positive data from the phase IIb AMPLIFIED study of its investigational drug inaxaplin in patients with APOL1-mediated kidney disease, or AMKD. In the first cohort of patients with AMKD and modest proteinuria, inaxaplin 45 mg once daily reduced urine albumin-to-creatinine ratio by 42.7% from baseline at week 13, while urine protein-to-creatinine ratio fell 44.7%. In the second cohort of patients with AMKD, type II diabetes and proteinuria, inaxaplin produced a 17.3% reduction in UACR and a 25.4% decline in UPCR at week 13. The drug was generally well-tolerated in both cohorts, with no serious adverse events reported. Vertex also said it has completed patient enrollment in the global phase II/III pivotal AMPLITUDE study of inaxaplin in patients with AMKD and severe proteinuria, and expects interim data in early 2027; positive results could potentially support accelerated approval in the United States, subject to regulatory review.

Novo Nordisk announced two European regulatory advances for its Rare Disease portfolio, with the European Medicines Agency's Committee for Medicinal Products for Human Use recommending approval of denecimig, to be marketed as Frehemgo, for hemophilia A in adults and children with or without inhibitors, and issuing a positive opinion for once-weekly Sogroya, or somapacitan, in children with idiopathic short stature or unexplained shortness. Both decisions still require final European Commission action. Novo expects to launch Frehemgo in the first European countries in the fourth quarter of 2026, followed by broader EU availability in early 2027, and denecimig is also under FDA review for the same hemophilia A indication. The Sogroya opinion follows a May 2026 CHMP recommendation for children with short stature born small for gestational age and those with Noonan syndrome, and the pending EC decision will cover all three indications; if approved, Sogroya would become the first and only growth hormone treatment authorized for idiopathic short stature in the EU. Novo generated DKK 9.1 billion in adjusted Rare Disease sales in the first half of 2026, and the segment also includes Alhemo, approved in the United States, Europe, Japan and Australia for routine prophylaxis in patients aged 12 years and older with hemophilia A or B, plus etavopivat, which has completed phase III development in sickle cell disease and phase II development in thalassemia. The Rare Disease push comes as Novo's core GLP-1 franchise faces intensifying competition, with Eli Lilly's Mounjaro generating $18.6 billion in first-half 2026 sales, up 106% year over year, and Zepbound sales rising 60% to $9.1 billion, while Novo's first-half Ozempic sales declined 2% at constant exchange rates and Wegovy product sales increased 7%.

The global autologous hematopoietic stem cell gene therapy market is projected to grow from $5.23 billion in 2025 to $6.30 billion in 2026, a compound annual growth rate of 20.6%, and to reach $13.44 billion by 2030 at a 20.8% CAGR, according to the Autologous Hematopoietic Stem Cell Gene Therapy Market Global Report 2026 added to ResearchAndMarkets.com. Growth drivers include commercialization of gene-editing therapies, rising regulatory approvals, wider adoption of personalized medicine and expanding investment in cell and gene therapy infrastructure, alongside next-generation non-viral delivery platforms. In February 2024, Vertex Pharmaceuticals received conditional marketing authorization from the European Commission for CASGEVY, developed with CRISPR Therapeutics, for patients aged 12 and older with severe sickle cell disease and transfusion-dependent beta thalassemia. In October 2025, AGC Biologics partnered with Rarity Public Benefit Corporation to support development and Good Manufacturing Practice manufacturing of RDP-101 for adenosine deaminase severe combined immunodeficiency. North America was the largest regional market in 2025, while Asia-Pacific is expected to be the fastest-growing region; featured companies include Novartis AG, Vertex Pharmaceuticals, CSL Behring, Orchard Therapeutics, CRISPR Therapeutics, Rocket Pharmaceuticals, Editas Medicine, Sangamo Therapeutics, Beam Therapeutics and Prime Medicine.

Japan's Ministry of Health, Labour and Welfare approved BioCryst Pharmaceuticals' once-daily ORLADEYO for children with hereditary angioedema aged 2 to 12, making it the first and only oral prophylactic option for pediatric HAE patients in the country. The clearance, announced August 25, expands on the drug's original 2021 Japanese approval for patients 12 and older and extends ORLADEYO's global footprint to more than 45 countries. In the US, where pellet shipments began August 3, prescribers wrote 47 scripts for children within days. BioCryst reported second-quarter revenue of $218.3 million, up 34% year over year, with GAAP operating profit of $98.5 million and $354.0 million in cash, though ORLADEYO net revenue grew just 1% on a reported basis as the total was lifted by $55.7 million from a European navenibart licensing deal. Full-year ORLADEYO guidance stayed at $625 million to $645 million, and Japanese commercial sales must still clear the National Health Insurance pricing process before launch.

Cantor Fitzgerald upgraded Omeros Corporation to Overweight from Neutral, citing bigger-than-anticipated sales prospects for its newly approved transplant therapy Yartemlea. Analyst Olivia Brayer Saunders also reinstated her price target of $22 on the biotech, whose shares set a new 52-week high on Tuesday. Omeros received FDA approval for Yartemlea in December for hematopoietic stem cell transplant-associated thrombotic microangiopathy, a complication of stem cell transplantation. Saunders raised her peak sales estimate for the drug to $400M, assuming 35% - 40% U.S. market share and no market share from Europe, and argued that $28.5M in Yartemlea sales recorded for Q2 was a huge number. Despite more than a 90% rally in Omeros shares over the past six months, she said the stock is pricing in no more than $350M in peak sales for the drug, adding that even after the move, investors are not fully giving Yartemlea credit for what this launch could become.

Stifel initiated coverage of Roivant Sciences with a Buy rating and a $51 price target on September 16, projecting roughly $9 billion in ownership-adjusted sales for the company by 2037. The call centers on Lisraya, or brepocitinib, which the FDA approved on August 27 for adults with dermatomyositis, making it the first FDA-approved oral treatment for the rare disease. Roivant is also evaluating brepocitinib in non-infectious uveitis, with Phase 3 topline data expected in the second half of 2026, in cutaneous sarcoidosis, with Phase 3 data expected in 2028, and in lichen planopilaris, where enrollment in Part 1 of a Phase 2b/3 study is on course. Beyond brepocitinib, the anti-FcRn antibody IMVT-1402 is advancing in several autoimmune indications with clinical updates expected in 2026, and mosliciguat posted positive Phase 2 results in pulmonary hypertension associated with interstitial lung disease, showing a 56.3% placebo-adjusted reduction in pulmonary vascular resistance at Week 16, prompting a Phase 3 program. Roivant reported $3.9 billion in cash, cash equivalents, restricted cash, and marketable securities at June 30, and in July Genevant and Arbutus received an aggregate $950 million payment from Moderna through a patent infringement settlement, with Genevant receiving $771.6 million. Hedge fund interest rose to 64 holders at the end of Q2 from 60 in Q1 and 52 in Q4, while short interest stood at 33.5 million shares as of August 31, equal to 5.09% of the public float.

The FDA on September 17 approved Fayuvi for the treatment of neurologic manifestations of Sanfilippo syndrome Type A in pediatric patients with preserved neurodevelopmental function, making it the first approved treatment for the rare and fatal childhood disorder. Ultragenyx Pharmaceutical Inc. expects Fayuvi to be available to patients within 30 to 60 days, and has set a list price of $3.95 million for a one-time treatment in the US, one of the world's most expensive drugs. JP Morgan estimates Fayuvi could bring in $200 million to $250 million in peak global sales, which at the lower end would represent around 26% of Ultragenyx's 2026 revenue guidance midpoint. The approval expands Ultragenyx's commercial portfolio as the company works toward profitability in 2027, after it posted a loss of $575 million on revenue of $673 million in 2025 and guided for $730 million to $760 million in total revenue this year from current products, excluding potential revenue from new launches such as Fayuvi. Ultragenyx stock jumped more than 12% on the approval but remains down nearly 40% this year, while short interest stood at 17.1 million shares, or 18% of the public float, as of August 31.

PTC Therapeutics has completed its previously announced agreement with Sangamo Therapeutics to acquire ST-920, a BLA-stage, one-time administered AAV gene therapy for Fabry disease. A rolling BLA submission to the FDA for accelerated approval of ST-920 is expected to be completed in the fourth quarter of 2026. Chief Executive Officer Matthew B. Klein said the company looks forward to completing the submission and potentially bringing a one-time administered, safe and effective durable disease treatment to the Fabry community. ST-920, also known as isaralgagene civaparvovec, has received Orphan Drug, Fast Track, and RMAT designations from the FDA, along with Orphan Medicinal Product designation and PRIME eligibility from the European Medicines Agency and Innovative Licensing and Access Pathway from the U.K. Medicines and Healthcare products Regulatory Agency. In clinical studies, the therapy enabled long-term production of the deficient alpha-galactosidase A enzyme and significant reduction in globotriaosylceramide levels.

AstraZeneca announced that the Committee for Medicinal Products for Human Use of the European Medicines Agency has issued a positive opinion recommending approval of its rare-disease drug Klygefa, also known as gefurulimab, as an add-on therapy for adults with generalised myasthenia gravis who are positive for anti-acetylcholine receptor antibodies. If approved, Klygefa would become the first and only dual-binding nanobody C5 inhibitor for that patient population in the European Union. The drug is already approved in Japan and certain other countries for specific adults with gMG, and is under regulatory review in the United States, China and some other countries for the same indication. The CHMP recommendation was supported by positive data from the pivotal phase III PREVAIL study, which met its primary endpoint by showing a treatment difference of -1.6 in the myasthenia gravis activities of daily living score at week 26 versus placebo, with clinically meaningful improvement seen as early as week one and sustained through week 26. About 85% of people living with gMG are AChR antibody-positive, and across five major European countries an estimated 82,500 people have gMG, including approximately 66,000 AChR antibody-positive patients.