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A clinician discusses Rett syndrome care with a patient and caregiver as Daybu moves closer to European approval following a positive CHMP recommendation. Representative image.
Pharma & Biotech

Acadia Pharmaceuticals secures positive CHMP opinion for Daybu in narrowed Rett syndrome indication

Discover why Daybu’s CHMP turnaround could reshape Rett syndrome treatment in Europe and what clinical and access hurdles remain.

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Viridian Therapeutics’ FDA-approved Lumvoa enters the thyroid eye disease market with a five-infusion treatment course, intensifying competition with Tepezza. Representative image.
Pharma & Biotech

FDA approves Lumvoa for thyroid eye disease as Viridian enters commercial biotech

See how Lumvoa’s FDA approval could reshape thyroid eye disease treatment, challenge Tepezza and test Viridian’s launch strategy.

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Representative image: An unbranded prefilled injection pen and blood samples illustrate PharmaEssentia’s FDA-approved BESREMi Pen launch for polycythemia vera treatment in the United States.
Pharma & Biotech

FDA approves BESREMi Pen as PharmaEssentia targets easier long-term polycythemia vera treatment

FDA approval brings BESREMi Pen to U.S. polycythemia vera care. Find out how convenience, safety and access may shape adoption.

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Representative image of clinicians reviewing cardiac and upper-limb assessments as Capricor prepares deramiocel for a decisive FDA advisory committee in Duchenne muscular dystrophy.
Pharma & Biotech

Capricor faces an FDA panel for deramiocel, but has HOPE-3 truly resolved its Duchenne evidence gap?

Capricor Therapeutics has secured a July 29 FDA advisory committee meeting for deramiocel after a positive Phase 3 trial revived the previously rejected Duchenne muscular dystrophy application. The therapy now has controlled evidence of skeletal and cardiac benefit, but regulators must still weigh endpoint interpretation, long-term external comparisons, manufacturing reliability and whether the proposed label

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Representative image of oncology researchers reviewing melanoma scans and treatment-response data as Replimune’s RP1 returns to the FDA for a decisive advanced melanoma approval review.
Pharma & Biotech

FDA gives Replimune’s RP1 a third chance, but can it overcome the same melanoma evidence gap?

The FDA has accepted Replimune’s latest RP1 resubmission and set an August 2 decision date, reviving a melanoma programme rejected twice over trial-design and efficacy questions. A late-July advisory committee will determine whether durable responses can outweigh the absence of definitive controlled evidence.

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Representative image of scientists reviewing liver biomarker and RNA-editing data as ProQR Therapeutics advances AX-0810 and AX-0811 in the race to develop a treatment for biliary atresia.
Pharma & Biotech

ProQR shows RNA editing works in humans, but can AX-0810 delay liver transplantation?

ProQR Therapeutics has produced the first human target-engagement evidence for its Axiomer RNA-editing platform. The results support its mechanism, but patient benefit, long-term safety and the choice between AX-0810 and AX-0811 remain unresolved.

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Representative image: Definium Therapeutics’ $805 million public offering strengthens its ability to advance DT120 ODT after positive Phase 3 depression trial results, while regulatory and commercial execution remain the next major tests.
Pharma & Biotech

DT120 delivered a Phase 3 surprise. Can Definium Therapeutics turn $805m into a launch?

Definium Therapeutics has used the strong market response to its DT120 ODT depression data to secure one of the largest recent biotechnology financings, giving the psychiatric drug developer substantial resources for additional trials, regulatory work and potential commercialisation.

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Representative image of researchers examining Alzheimer’s disease biomarker data as NeuroSense evaluates PrimeC’s multi-target potential in a small exploratory study.
Pharma & Biotech

NeuroSense links PrimeC to Alzheimer’s biomarkers, but can three patients justify another trial?

NeuroSense Therapeutics says PrimeC produced biomarker changes consistent with its multi-target mechanism in an exploratory Alzheimer’s study, but an exceptionally small dataset leaves clinical efficacy, safety and commercial relevance unanswered.

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A clinician reviews an investigational treatment as MaaT Pharma prepares to challenge the negative European regulatory opinion on MaaT013 for acute graft-versus-host disease. Representative image.
Pharma & Biotech

MaaT Pharma seeks second review of MaaT013 as regulators question single-arm evidence

Discover why MaaT Pharma is challenging the negative CHMP opinion on MaaT013 and what the September review means for acute graft-versus-host disease.

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Researchers review mechanistic AI models for drug safety and liver toxicity assessment as the VeriSIM Life and FDA/NCTR collaboration explores more transparent, human-relevant drug development tools. Representative image.
Pharma & Biotech

VeriSIM Life targets drug development’s translation problem with FDA/NCTR research pact

See how VeriSIM Life’s FDA/NCTR research collaboration could shape transparent mechanistic AI for safer, faster drug development.

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