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Confusion grows amid uniQure and FDA’s AMT-130 row

Dutch biotech uniQure closed Friday’s trading 34% higher - and was up by a similar amount again in pre-market Monday - as the chaos and confusion intensified around AMT-130, its investigational gene therapy for Huntington’s disease (HD). 9 March 2026

The week in pharma: action, reaction and insight – week to March 6, 2026

Significant research news last week included United Therapeutics releasing positive Phase III data on its ralinepag for pulmonary arterial hypertension (PAH). French biotech OSE Immunotherapeutics announced a strategic refocusing of its pipeline. On the deal making front, Moderna reached a $2.25 billion settlement with Genevant and Arbutus on authorized use of CNP technology. The week ended with the Director of the US Center for Biologics Evaluation and Research, Dr Vinay Prasad, saying he would be leaving the post in April. 8 March 2026

CBER chief Vinay Prasad to quit

After the close of stock markets on Friday, the Wall street Journal (WSJ) reported (here) that Director of the Center for Biologics Evaluation and Research (CBER), Dr Vinay Prasad, will leave the agency in April. 7 March 2026

Sanofi licenses rovadicitinib from Sino Biopharm in up to $1.5 billion deal

French pharma Sanofi has agreed to license the experimental drug rovadicitinib from China’s Sino Biopharmaceutical, paying $135 million upfront for exclusive development and commercialization rights outside China. 5 March 2026

Candid lists via Rallybio reverse merger, adds $505m financing

US biotechnology company Candid Therapeutics will go public through a reverse merger with US rare disease drugmaker Rallybio, creating a Nasdaq-listed company focused on T-cell engager therapies for autoimmune diseases. 4 March 2026

Share tumbles and jobs to go after Theravance failure

Ireland-incorporated Theravance Biopharma has announced top-line results from the Phase III CYPRESS study of ampreloxetine in patients with symptomatic neurogenic orthostatic hypotension (nOH) due to multiple system atrophy (MSA), a rare disease. 4 March 2026

uniQure shares slide on FDA demands for new gene therapy trial

Dutch biotech uniQure has announced the receipt of final minutes from the US Food and Drug Administration (FDA) regarding a Type A meeting held on January 30 to discuss AMT-130, its investigational gene therapy for Huntington’s disease (HD). 3 March 2026

FDA grants Takeda and Protagonist’s rusfertide priority review

The US Food and Drug Administration (FDA) accepted the New Drug Application (NDA) and granted Priority Review for rusfertide. 2 March 2026

February 2026 rare disease industry highlights

February 2026 delivered significant signals across the rare disease ecosystem, spanning regulatory approvals, legislative incentives, clinical trial outcomes, emerging designations, and safety reviews. Below is a summary of the most impactful developments. 2 March 2026

FDA nod for BioMarin’s Palynziq in adolescents

BioMarin Pharmaceutical today announced that the US Food and Drug Administration (FDA) has approved the company’s supplemental Biologics License Application (sBLA) for Palyvziq (pegvaliase-pqpz) to include pediatric patients 12 years of age and older with phenylketonuria (PKU). 28 February 2026

Santhera’s Agamree wins Spanish reimbursement

Switzerland’s Santhera Pharmaceuticals has announced that the Spanish Interministerial Commission on Prices of Medicines has proposed to include Agamree (vamorolone) in the pharmaceutical coverage of the National Health System, making it eligible for reimbursement. 27 February 2026

February batch of novel medicines recommended for approval by EMA/CHMP

The European Medicines Agency’s (EMA) human medicines committee (CHMP) recommended 6 novel medicines for approval at its February 2026 meeting. These now have to be approved by the European Commission, which usually takes two months or so. 27 February 2026

Sarepta seeks new boss amid Doug Ingram retirement

Douglas Ingram has told Sarepta Therapeutics of his decision to retire as chief executive of the US genetic medicines company by the end of 2026 or upon the appointment of his replacement. 26 February 2026

UCB posts strong fourth quarter finish as profit surges

Belgian biopharma group UCB closed 2025 with fourth-quarter momentum that capped a year of sharp growth, reporting full-year revenue of 7.7 billion euros ($8.4 billion), up 26%, and core earnings per share of 9.99 euros, compared with 4.98 euros in 2024. 26 February 2026

Neurizon doses first patient with NUZ-001 in US HEALEY ALS trial

Australian biotech Neurizon has dosed the first participant with NUZ-001 in Regimen I of the US-based HEALEY ALS Platform Trial, launching a key Phase II/III study in amyotrophic lateral sclerosis. 26 February 2026

Gossamer shares plunge after Phase III miss for seralutinib in PAH

US biotech Gossamer Bio saw its shares collapse 81% after its Phase III PROSERA study of seralutinib in pulmonary arterial hypertension (PAH) missed a prespecified statistical bar, denting hopes of challenging Merck & Co’s (NYSE: MRK)Winrevair (sotatercept). 24 February 2026

Chugai launches Elevidys as Japan’s first regenerative med for DMD

Japan’s Chugai today announced that it launched Elevidys intravenous infusion (delandistrogene moxeparvovec), Japan’s Chugai (TYO: 4519) today announced that it launched Elevidys intravenous infusion (delandistrogene moxeparvovec), which received conditional and time-limited approval on May 13, 2025, following its listing on the national health insurance (NHI) reimbursement price list today, as a regenerative medical product for the treatment of Duchenne muscular dystrophy (DMD) in Japan.following its listing on the national health insurance (NHI) reimbursement price list today, as a regenerative medical product for the treatment of Duchenne muscular dystrophy (DMD) in Japan. 20 February 2026

Hansa Biopharma’s BLA for imlifidase accepted by the FDA

Swedish biotech Hansa Biopharma late yesterday announced that its Biologics License Application (BLA) for imlifidase has been accepted by the US Food and Drug Administration (FDA). 19 February 2026

Recordati delivers solid year, with more to come in 2026

Italian pharma major Recordati has reported 2025 revenues of 2.62 billion euros ($3.1 billion), which was in line with analyst expectations and up 11.8% versus full year 2024. 18 February 2026

Rare Disease Day: Digital twins offer hope for patients

Rare Disease Day on February 28, highlights the persistent challenges of developing treatments for conditions where patient numbers are small and data is scarce. 18 February 2026

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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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