Rare Diseases
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Biotechnology
US late-stage biotech Immunic yesterday announced the closing of its private placement financing. 18 February 2026
Biotechnology
US rare disease specialist Ultragenyx Pharmaceutical is cutting around 130 employees, or 10% of its workforce, as part of a restructuring plan aimed at reaching profitability in 2027. 16 February 2026
Biotechnology
Last week’s news included US pharma major Eli Lilly revealing an up to $2.4 billion deal to acquire Orna Therapeutics to expand its cell therapies pipeline. On the regulatory front, Regenxbio announced it had received a complete response letter (CRL) from US the Food and Drug Administration (FDA) regarding its Hunter syndrome candidate RGX-121, and mRNA specialist Moderna received a refuse to file letter from the FDA relating to its flu vaccine mRNA-1010. Also of note, BridgeBio released positive new data on its achondroplasia candidate infigratinib. 15 February 2026
Pharmaceutical
USA-based rare disease drug developer PTC Therapeutics saw its shares drop 5.2% to $67.05 after market close, when it announced that it has withdrawn the New Drug Application (NDA) resubmission for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD) following US Food and Drug Administration (FDA) feedback on the application review. 13 February 2026
Biotechnology
US biotech giant Amgen today announced the European Commission (EC) has approved Uplizna (inebilizumab) as an add-on treatment to standard therapy for adults living with generalized myasthenia gravis (gMG). 12 February 2026
Pharmaceutical
For the entire 2025 fiscal year, French drugmaker Ipsen’s revenues rose by 8.1%, or 10.9% at constant exchange rates, reaching a total of 3.68 billion euros ($4.37 billion). 12 February 2026
Biotechnology
France’s THX Pharma, formerly known as Theranexus and specializing in the treatment of rare neurological diseases, and Biocodex, an independent international pharmaceutical group, announce the execution of a strategic licensing agreement to advance the development of two drug candidates in three rare diseases with high unmet medical needs: Batten disease with Batten-1, and Gaucher disease and Niemann-Pick disease type C with TX01. 11 February 2026
Pharmaceutical
British pharma major AstraZeneca has reported fourth-quarter revenue of $15.5 billion, up 4%, with reported earnings per share of $1.50, a 55% increase. Core EPS was $2.12, broadly flat year on year. 10 February 2026
Biotechnology
Shares in US gene therapy developer Regenxbio slid more than 17% in after-hours trading on Monday after the American medicines regulator issued a complete response letter for RGX-121, its experimental treatment for mucopolysaccharidosis type II, also known as Hunter syndrome. 10 February 2026
Pharmaceutical
The US Food and Drug Administration (FDA) has granted a designation as breakthrough therapy to Wayrilz (rilzabrutinib), a novel oral, reversible Bruton’s tyrosine kinase (BTK) inhibitor, for the treatment of patients with warm autoimmune hemolytic anemia (wAIHA), a rare autoimmune disorder marked by the destruction of red blood cells, French pharma major Sanofi announced this morning. 9 February 2026
Pharmaceutical
Swiss pharma giant Roche has announced new late-breaking data from the Phase III FENtrepid study showing the investigational Bruton’s tyrosine kinase (BTK) inhibitor fenebrutinib met its primary endpoint of non-inferiority compared to Ocrevus (ocrelizumab) in reducing disability progression in patients with primary progressive multiple sclerosis (PPMS). 9 February 2026
Pharmaceutical
Health Canada has authorized Livmarli (maralixibat) oral solution and tablet formulation for the treatment of cholestatic pruritus in patients aged 12 months or older with progressive familial intrahepatic cholestasis (PFIC), Mirum Pharmaceuticals announced on Friday. 7 February 2026
Biotechnology
US biotech company Biogen today reported fourth quarter 2025 results topping the market’s revenue expectations, but sales fell by 7.1% year on year to $2.28 billion. Biogen’s share were up 4.3% at $192.97 in early trading. 6 February 2026
Biotechnology
Swiss biotech firm Idorsia today announced the design of its US Food and Drug Administration (FDA)-agreed Phase III registration program for lucerastat in Fabry disease. 6 February 2026
Biotechnology
US biotech major Amgen announced financial results for the fourth quarter and full year of 2025 after markets closed on Tuesday. 4 February 2026
Biotechnology
US biotech Wave Life Sciences has regained full rights to WVE-006 from British pharma GSK, bringing the RNA-editing therapy for alpha-1 antitrypsin deficiency back in-house as it prepares for higher-dose clinical data and steps up regulatory engagement. 3 February 2026
Pharmaceutical
French pharma Sanofi says Phase III results for its oral candidate venglustat improved neurological symptoms in people with type 3 Gaucher disease, a rare inherited disorder. Patients aged 12 years and older in the LEAP2MONO study met the primary endpoint and three of four key secondary endpoints. 2 February 2026
Pharmaceutical
The European Medicines Agency’s (EMA) human medicines committee (CHMP) has started a review of Amgen’s Tavneos (avacopan), following emerging information that raises questions regarding the data integrity of the Advocate study. 2 February 2026
Pharmaceutical
US biotech BioMarin Pharmaceutical has priced an $850 million offering of 5.5% senior unsecured notes due 2034, strengthening its financing package for the planned acquisition of Amicus Therapeutics. 2 February 2026
Pharmaceutical
Dutch biopharma company Pharming Group saw its shares tumble 13.5% to 1.48 euros this morning after it received a regulatory setback. 2 February 2026
Pharmaceutical
New data published in Brain Communications offers a strong picture of how devastating thymidine kinase 2 deficiency (TK2d) can be without treatment - and how vital earlier diagnosis and treatment are in potentially changing the course of disease. 17 June 2026
Biotechnology
Polish biotech Mabion has signed a letter of intent with French TechDev company Oddifact to explore MabionCD20 in rare diseases, reopening a program that was once developed as a rituximab biosimilar. 17 June 2026
Biotechnology
San Diego, USA-based Soleno Therapeutics, which has just been acquired by Neurocrine Biosciences, has announced late-breaking data at ENDO 2026 showing that resuming treatment with Vykat XR (diazoxide choline) extended-release tablets . 16 June 2026
Biotechnology
Shares of Australian biotech Dimerix leapt 21.2% to A$0.20 today on the news of a landmark licensing deal. 16 June 2026
Biotechnology
China-based Fosun Pharmaceutical announced that its self-developed MEK 1/2 selective inhibitor luvometinib tablets (trade name: Fumaining) has been officially approved by China’s National Medical Products Administration (NMPA). 16 June 2026
Pharmaceutical
US biopharma Neurocrine Biosciences has reported new two-year data for Crenessity (crinecerfont), showing sustained reductions in disease biomarkers, lower glucocorticoid dosing and improvements in clinical measures among children and adolescents with classic congenital adrenal hyperplasia (CAH). 15 June 2026
Biotechnology
US CRISPR gene editing company Intellia Therapeutics over the weekend presented additional positive results from the global Phase III HAELO clinical trial of lonvoguran ziclumeran (lonvo-z; formerly NTLA-2002) for hereditary angioedema (HAE). 15 June 2026
Biotechnology
M&A featured heavily last week, with Incyte announcing plans to buy Vega Therapeutics, a subsidiary of Star Therapeutics, for up to $2 billion. Then came UK pharma major GSK’s revelation of an agreement to acquire US firm Nuvalent for up to $10.6 billion. Also of note, US biotech Cartesian Therapeutics entered into a licensing deal with China’s WestGene Biopharma on CAR-T therapies for autoimmune diseases. Sweden’s Camurus was served another complete response letter (CRL) by the US Food and Drug Administration (FDA) on its filing for Oclaiz (CAM2029). 14 June 2026
Pharmaceutical
Shares in Sweden based drug developer Camurus fell as much as 7% on Thursday, after the US Food and Drug Administration (FDA) rejected the company’s application for Oclaiz (octreotide; CAM2029) extended-release injection a treatment for acromegaly. 12 June 2026
Biotechnology
Swiss pharma Novartis said its investigational RNA therapy del-brax has met the primary and key secondary endpoints in a Phase I/II study of facioscapulohumeral muscular dystrophy, supporting the dose already being tested in a Phase III trial. 12 June 2026
Pharmaceutical
An interview with Anna Petryk, Executive Global Medical Director who leads the global medical affairs team in the Rare Bone and Endocrine Disorders therapeutic area at Alexion, AstraZeneca Rare Disease. 11 June 2026
Biotechnology
US biotech Beren Therapeutics has lined up a financing package worth up to $300 million to support preparations for the potential launch of adrabetadex, its investigational treatment for infantile-onset Niemann-Pick disease type C. 11 June 2026
Biotechnology
San Francisco-based biotech SonoThera today announced the closing of an oversubscribed $125 million Series B financing round to advance its lead programs in Duchenne muscular dystrophy (DMD) and autosomal dominant polycystic kidney disease (ADPKD) into the clinic and expand its pipeline of targeted, redosable genetic medicines. 10 June 2026
Pharmaceutical
French drugmaker Servier has entered a strategic collaboration with the USA-based n-Lorem Foundation to advance research into rare neurodevelopmental disorders using antisense oligonucleotide (ASO) technology. 10 June 2026
Biotechnology
Swedish rare diseases company Hansa Biopharma today announced that Fredrik Röök has been appointed as vice president business development effective August 17. 10 June 2026
Pharmaceutical
The European Federation of Pharmaceutical Industries and Associations (EFPIA) has called for stronger action on rare diseases, warning that fragmented systems continue to hold back research, diagnosis and access to treatment. 8 June 2026
Pharmaceutical
Among licensing news of note last week, US drug developer Agios Pharma struck a deal with South Korea’s Oscotec for the latter’s cevidoplenib, a potential treatment for immune thrombocytopenia. US major AbbVie won European Commission approval for its migraine drug Aquipta. Travere Therapeutics licensed rights to civorebrutinib from China’s Everest Medicines, and Alnylam Pharmaceuticals entered into an AI collaboration with Inceptive Nucleics worth a potential $2 billion. 7 June 2026
Pharmaceutical
Chiesi Global Rare Diseases, a unit of privately-held Italian drugmaker Chiesi, announced that the European Commission (EC) has approved Lojuxta (lomitapide) capsules for use in children 5 years of age and older with homozygous familial hypercholesterolemia (HoFH), for use alongside diet and other lipid-lowering treatments, including LDL-apheresis where available. 6 June 2026
Generics
Russia will have its own generic of one of the world’s most expensive drug against cystic fibrosis, as the Russian pharmaceutical company Amedart announced its plans launch a domestic analogue of Trikafta (elexacaftor/tezacaftor/ivacaftor), a drug from the American company Vertex Pharmaceuticals for the treatment of cystic fibrosis, reports The Pharma Letter’s local correspondent. 5 June 2026
Biotechnology
US biotech Biogen has received US Food and Drug Administration (FDA) Breakthrough Therapy designation for salanersen, its investigational antisense oligonucleotide for spinal muscular atrophy (SMA). 5 June 2026
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