Ultragenyx Receives FDA Accelerated Approval for GENGLYCOS Gene Therapy to Treat the Underlying Cause of GSDIa

Shots: Ultragenyx reported the US FDA accelerated approval to GENGLYCOS (pariglasgene brecaparvovec-opnr; DTX401) for adults and pediatric patients (≥8yrs., n=46) with glycogen storage disease type Ia (GSDIa) The approval was supported by the P-III (GlucoGene) study, in which GENGLYCOS (n=20) demonstrated a statistically significant reduction in cornstarch requirements at 48wks. vs PBO (n=24) (p<0.001). The…

ByPrince GiriAugust 20, 2026

NEWS

Alfasigma to Acquire Nordic Pharma, Expanding Rheumatology and Global Reach

Shots: Alfasigma has entered into an agreement with SEVER Life Sciences to acquire 100% of Nordic Pharma & its subsidiaries, adding the Nordimet methotrexate franchise to Alfasigma’s rheumatology portfolio alongside Jyseleca Deal will expand Alfasigma’s commercial footprint through Nordic Pharma’s direct operations across 18 EU countries, while adding platforms in Canada & Japan. The portfolio…

ByRidhi RastogiAugust 11, 2026

NEWS

Chiesi Group and Arbor Biotechnologies Secure the EC’s Orphan Drug Designation for ABO-101 to Treat Primary Hyperoxaluria

Shots: The EC has granted ODD to ABO-101 for the treatment of primary hyperoxaluria ABO-101 is being evaluated in an open-label global P-I/II (redePHine) study assessing safety, tolerability, PK/PD, & preliminary efficacy of a single dose of ABO-101 in pts with PH type 1 ABO-101 is a one-time, liver-directed gene-editing therapy that uses an Acuitas-licensed…

ByRidhi RastogiJune 26, 2026

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Advancing HES Care with AstraZeneca’s FASENRA: Insights from James Teague & Princess U. Ogbogu

Shots: Could a targeted biologic therapy help address the longstanding unmet needs in hypereosinophilic syndrome (HES)? AstraZeneca’s FASENRA has now received US approval for HES, introducing a once-monthly treatment option for patients living with this rare and often underdiagnosed eosinophilic disorder Results from the Phase III NATRON trial showed that FASENRA reduced the risk of first HES flare by 65% vs placebo,…

BySaurabh ChaubeyJune 1, 2026

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CVC Capital and Groupe Bruxelles Lambert (GBL) Launch €10.7B Tender Offer to Take Recordati Private

Shots: A consortium led by CVC and GBL launched a voluntary cash tender offer to acquire Recordati at €51.29/share, valuing the transaction at ~€10.7B (~$12.4B). The offer aims to delist Recordati from Euronext Milan & is expected to close in Q4’26 The offer is backed by Rossini, which agreed to tender its 97.9M shares (~46.8%…

ByRidhi RastogiMay 25, 2026

NEWS

Angelini Pharma to Acquire Catalyst Pharmaceuticals for ~$4.1B

Shots: Angelini Pharma has entered into a definitive agreement to acquire all outstanding shares of Catalyst, marking Angelini’s entry into the US market As per the deal, Angelini will acquire Catalyst for $31.5 per share in cash, for a total equity value of ~$4.1B; closing is expected in Q3’26 The acquisition will strengthen Angelini’s Brain Health…

ByRidhi RastogiMay 7, 2026

NEWS

X4 Pharmaceuticals’ Xolremdi Receives the EC’s Approval for WHIM Syndrome

Shots: The EC has approved Xolremdi (Mavorixafor), under exceptional circumstances, for the treatment of WHIM syndrome Approval was supported by the global pivotal P-III (4WHIM) trial, PBO-controlled 52-wk study evaluating mavorixafor’s efficacy & safety in pts ≥12 yrs with WHIM syndrome Xolremdi (PO, QD), a CXCR4 antagonist, is used in pts ≥12 yrs with WHIM syndrome to increase circulating mature neutrophils & lymphocytes Ref: X4…

ByDipanshu DixitApril 27, 2026

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Crinetics Pharmaceuticals Report the EC Approval of Palsonify for Acromegaly

Shots: The EC has approved Palsonify (paltusotine) for treating adults with acromegaly who are ineligible for or inadequately respond to surgery across all 30 EEA states EC approval was supported by P-III PATHFNDR-1 & PATHFNDR-2 trials evaluating Palsonify in previously treated & medically untreated adults with acromegaly, demonstrating rapid onset, reliable biochemical control & sustained efficacy; it also received ODD in EU Crinetics plans…

ByDipanshu DixitApril 23, 2026

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Novo Nordisk Reports Positive P-III (HIBISCUS) Study Data for Etavopivat in Sickle Cell Disease

Shots: Novo Nordisk reported that etavopivat met both co-1EPs in the P-III (HIBISCUS) study, reducing vaso-occlusive crises (VOCs) and improving haemoglobin response in sickle cell disease The study (n=385, ≥12yrs) evaluated Etavopivat (400mg) vs PBO demonstarting reduced VOCs rates by 27% and delayed time to first VOC (~38.4 vs 20.9 weeks), while 48.7% of patients…

ByPrince GiriApril 20, 2026

NEWS

Sobi Reports Health Canada Approval of Empaveli for C3G and Primary IC-MPGN

Shots: Health Canada has approved Empaveli (pegcetacoplan) for patients (≥12yrs.) with C3 glomerulopathy (C3G) or primary IC-MPGN to reduce proteinuria, marking the first approved therapy for these indications in Canada Approval was based on the P-III (VALIANT) study, where Empaveli demonstrated a 68% reduction in proteinuria (uPCR), stabilization of kidney function, and clearance of C3…

ByPrince GiriApril 10, 2026

Ultragenyx Receives FDA Accelerated Approval for GENGLYCOS Gene Therapy to Treat the Underlying Cause of GSDIa

Alfasigma to Acquire Nordic Pharma, Expanding Rheumatology and Global Reach

Chiesi Group and Arbor Biotechnologies Secure the EC’s Orphan Drug Designation for ABO-101 to Treat Primary Hyperoxaluria

Advancing HES Care with AstraZeneca’s FASENRA: Insights from James Teague & Princess U. Ogbogu

CVC Capital and Groupe Bruxelles Lambert (GBL) Launch €10.7B Tender Offer to Take Recordati Private

Angelini Pharma to Acquire Catalyst Pharmaceuticals for ~$4.1B

X4 Pharmaceuticals’ Xolremdi Receives the EC’s Approval for WHIM Syndrome

Crinetics Pharmaceuticals Report the EC Approval of Palsonify for Acromegaly

Novo Nordisk Reports Positive P-III (HIBISCUS) Study Data for Etavopivat in Sickle Cell Disease

Sobi Reports Health Canada Approval of Empaveli for C3G and Primary IC-MPGN

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