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Over 90% reduced death risk with treatment in subgroup for ultra rare TK2d disease

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

Mabion turns to Oddifact in rare disease push for CD20 asset

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

Soleno Therapeutics presents new Vykat XR data at ENDO

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

Dimerix out-licenses DMX-200 to Everest for Asian markets

Shares of Australian biotech Dimerix leapt 21.2% to A$0.20 today on the news of a landmark licensing deal. 16 June 2026

Fosun Pharma’s innovative drug Fumaining approved in China

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

Two-year data bolster Neurocrine’s Crenessity case in pediatric CAH

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

Positive Phase III results for Intellia’s lonvo-z

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

The week in pharma: action, reaction and insight – week to June 12, 2026

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

FDA issues another CRL on Camurus NDA for CAM2029 in acromegaly

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

Novartis FSHD drug meets key biomarker goals in Phase I/II study

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

From raising awareness to developing treatments, Alexion is fighting HPP battle on many fronts

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

Beren lines up $300 million ahead of potential adrabetadex launch

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

SonoThera closes $125 million Series B financing

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

Servier partners with n-Lorem to advance rare neurology research

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

Hansa Biopharma names Fredrik Röök as VP business development

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

EFPIA urges stronger EU action on rare diseases

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

The week in pharma: action, reaction and insight – week to June 5, 2026

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

EC nod for Chiesi’s Lojuxta for pediatric HoFH patients

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

Russia aims to have generic of one of the most expensive cystic fibrosis drugs

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

Biogen’s salanersen gets Breakthrough Therapy designation in SMA

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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Relay Therapeutics rises on positive data for zovegalisib

Shares of US precision medicines company Relay Therapeutics were up 11.3% at $12.10 in pre-market activity today, after it announced highly encouraging initial clinical data from the Phase ReInspire trial of zovegalisib in vascular anomalies signaling the advantage of PI3Kα mutant-selective inhibition. 19 May 2026

Immunic appoints Michael Bonney as chairman

US biotech Immunic today announced the appointment of Michael "Mike" W Bonney, a highly experienced biopharmaceutical executive and board leader, as chair of its board of directors, effective May 16, 2026. 19 May 2026

NICE backs Winrevair for PAH

Merck & Co—the US pharma giant known as MSD outside North America—has welcomed a recommendation from the National Institute for Health and Care Excellence (NICE) supporting the use of Winrevair (sotatercept) for certain adults with pulmonary arterial hypertension (PAH) in England and Wales. 15 May 2026

Chiesi wins UK approval for monthly Elfabrio dosing

15 May 2026

Recordati beats on profit growth as rare disease drugs lift first-quarter sales

Italian drugmaker Recordati reported first-quarter 2026 revenue of 713 million euros ($775 million) on Tuesday, up 5% year-on-year, while diluted earnings per share rose 22% to 0.73 euros, driven by continued growth in its rare diseases business. 13 May 2026

AOP Health and VRG Thera partner on new immunology program

AOP Health, an Austrian enterprise group focused on integrated therapies for rare diseases and critical care, today announced a strategic partnership with VRG Therapeutics, an AI-driven developer for miniproteins based in Budapest, Hungary. 12 May 2026

Capricor sues over alleged NS Pharma inaction on deramiocel

San Diego-based Capricor Therapeutics, a biotech developing cell and exosome-based therapeutics for rare diseases, has filed a lawsuit Japanese pharma against Nippon Shinyaku and its US subsidiary, NS Pharma. 11 May 2026

Nxera progresses out-licensing of GPCR-targeted program

Japanese drugmaker Nxera Pharma has entered into a license agreement with a newly established company (NewCo) to advance a specific GPCR-targeted program owned by Nxera. Nxera shares rose 6.4% to 1,242 yen on the news. 11 May 2026

FDA expands approval of argenx’ VyvgartFDA expands approval of argenx’ Vyvgart

The US Food and Drug Administration (FDA) late Friday approved a label expansion for Netherlands-based argenx’ Vyvgart efgartigimod alfa-fcab) and Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) for the treatment of adult patients with generalized myasthenia gravis (gMG). 9 May 2026

Italfarmaco’s new CEO and commitment to rare disease R&D

An interview with Francesco Di Marco, the new chief executive of Italfarmaco. 8 May 2026

MHRA approves Otsuka’s Dawnzera

The UK Medicines and Healthcare products Regulatory Agency (MHRA) has today approved Japanese drugmaker Otsuka’s Dawnzera (donidalorsen) to prevent angioedema attacks. 7 May 2026

Angelini Pharma to acquire Catalyst Pharmaceuticals

Shares of Catalyst Pharmaceuticals are trading at around $30.52 to $31.50, following an announcement that Italy’s Angelini Pharma will acquire the company for $31.50 per share in cash. 7 May 2026

Takeda positions TAK-881 as next-gen option for PID patients

Japanese drugmaker Takeda said its investigational immunoglobulin therapy TAK-881 met the primary endpoint in a pivotal Phase II/III study in patients with primary immunodeficiency disease, strengthening the company’s position in a steadily growing plasma therapies market. 6 May 2026

UCB to acquire Candid Therapeutics

Adding to recent deal and M&S activity, Belgium’s largest drugmaker UCB over the weekend announced signing of a definitive agreement under which it would acquire US biotech Candid Therapeutics. 5 May 2026

Vertex beats on profit as revenue misses, shares dip

US biotech Vertex Pharmaceuticals reported first-quarter 2026 revenue of $3.0 billion, up 8%, while diluted EPS rose to $4.02 from $2.49 a year earlier. The company beat on earnings but missed revenue expectations, with shares slipping 0.9% after hours. 5 May 2026

The week in pharma: action, reaction and insight – week to May 1, 2026

US biopharma company Veradermics last week reported encouraging top-line Phase II/III results for its hair loss treatment VDPHL01. On the M&A front, Eli Lilly announced it is to acquire Ajax Therapeutics for up to $2.3 billion along with its hematology candidate AJ1-110915. Also, Chiesi announced a takeover of USA-based KalVista Pharma and its Ekterly to expand its rare diseases portfolio. US biotech AnaptysBio won a favorable court ruling in its dispute with GSK/Tesaro over cancer drug Jemperli. 3 May 2026

Pharmacological advances in rare diseases

April 2026 marked a period of cautious progress in rare disease therapeutics. A series of regulatory approvals, research developments, and policy initiatives signalled continued momentum, particularly in pediatric nephrology and oncology. 2 May 2026

Amgen 1st-qtr 2026 financials beat expectations

US biotech major Amgen late yesterday announced financial results for the first quarter of 2026, showing that revenues increased 6% to $8.6 billion in comparison to the first quarter of 2025, surpassing the Zacks consensus estimate by 1.71%. 1 May 2026

UK nod for Sobi’s Aspaveli for C3G and primary IC-MPGN

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has approved Aspaveli (pegcetacoplan) to treat patients aged 12 to 17 years with C3 glomerulopathy (C3G) or primary immune- complex membranoproliferative glomerulonephritis (IC-MPGN) in combination with a renin-angiotensin system (RAS) inhibitor, unless RAS inhibitor treatment is not tolerated or contraindicated. 1 May 2026

Hemab Therapeutics sets terms for $200 million IPO

Hemab Therapeutics Holdings, a Phase III-ready biotech developing blood coagulation disorder treatments, has announced terms for its initial public offering (IPO). 1 May 2026

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