AstraZeneca Reports FDA Approval of Saphnelo for Systemic Lupus Erythematosus (SLE)

Shots: The US FDA has approved AstraZeneca’s Saphnelo pen (anifrolumab; 120mg, SC, QW, autoinjector) for the treatment of adults with SLE in combination with SoC Approval was based on the P-III (TULIP-SC) trial assessing Saphnelo pen vs PBO in 367 pts (18-70yrs.) with mod. to sev. SLE while receiving SoC, which showed Saphnelo reduced disease activity (1EP); full…

ByRidhi RastogiApril 27, 2026

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Tanabe Pharma Report the EC Approval of Onerji for Parkinson’s Disease (PD)

Shots: The EC has approved Onerji (levodopa/carbidopa) infusion for advanced PD pts with uncontrolled motor fluctuations. The therapy is being developed by its wholly owned subsidiary, NeuroDerm Approval was based on the global P-III (BouNDless) trial, which demonstrated Onerji + supplemental oral LD/CD significantly increased ON time without troublesome dyskinesia and reduced OFF time vs immediate-release oral LD/CD. Favourable long-term safety and tolerability results were also observed in the…

ByDipanshu DixitApril 27, 2026

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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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Sanofi Reports the CHMP Positive Opinion for Cenrifki (Tolebrutinib) to Treat Non-Relapsing SPMS

Shots: The CHMP has recommended Sanofi's Cenrifki for the treatment of SPMS without relapses in the last 2yrs.; regulatory submissions are ongoing in other regions Opinion was backed by P-III (HERCULES) trial in non-relapsing SPMS & supporting data from P-III (GEMINI 1 & 2) studies in relapsing multiple sclerosis, with HERCULES demonstrating delayed onset of…

ByRidhi RastogiApril 27, 2026

NEWS

Novartis’ Itvisma Receives the CHMP Positive Opinion for Spinal Muscular Atrophy

Shots: The CHMP has recommended Novartis' Itvisma (onasemnogene abeparvovec) for the treatment of pts (≥2yrs.) living with 5q spinal muscular atrophy (SMA) harboring a confirmed bi-allelic mutation in the SMN1 gene Opinion was based on the P-III (STEER) trial & P-IIIb (STRENGTH) study, where STEER showed a 2.39-point improvement in the HFMSE with effects sustained…

ByRidhi RastogiApril 27, 2026

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Arrowhead Pharmaceuticals Receives the CHMP’s Positive Opinion for Redemplo (Plozasiran) to Treat Familial Chylomicronemia Syndrome (FCS)

Shots: The CHMP has recommended Arrowhead's Redemplo as an adjunct to diet to reduce triglycerides (TGs) in adults with FCS; EC’s decision is expected in Q2’26 Opinion was supported by the P-III (PALISADE) trial assessing Redemplo (25mg & 50mg, SC, Q3M) vs PBO in 75 adults with genetically confirmed/clinically diagnosed FCS. Pts could join a…

ByRidhi RastogiApril 24, 2026

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Regeneron Reports the US FDA Accelerated Approval of Otarmeni (lunsotogene parvec-cwha) in Genetic Hearing Loss

Shots: Otarmeni was approved under CNPV for pediatric & adult pts with sev. to profound and profound sensorineural hearing loss due to biallelic OTOF gene variants, with preserved outer hair cell function & no prior cochlear implant. It will be provided free of charge in the US by Regeneron Approval was based on the P-I/II…

ByRidhi RastogiApril 24, 2026

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DemeRx Reports the US FDA IND Application Acceptance to Advance DMX-1001 for Alcohol Use Disorder

Shots: The US FDA has accepted IND application of DMX-1001 (oral noribogaine) for the treatment of alcohol use disorder (AUD) DemeRx completed a MAD trial of DMX-1001 in healthy volunteers, demonstrating safety, tolerability, & PK supportive of P-II in AUD. The company plans to initiate a P-II trial of DMX-1001 in AUD in 2027 Noribogaine…

ByRidhi RastogiApril 23, 2026

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Regeneron and Sanofi Report the US FDA Approval of Dupixent (Dupilumab) for Chronic Spontaneous Urticaria (CSU)

Shots: FDA has approved Dupixent to treat CSU pts (age 2-11yrs.), who were symptomatic despite H1 antihistamine treatment based on 2 P-III (Study A & C) trials from LIBERTY-CUPID program, & P-III (CUPIDKids) trial Study A & C assessed Dupixent + SoC antihistamines (loading dose then 300mg Q2W; pts weighing <60kg received 200mg) vs PBO…

ByRidhi RastogiApril 23, 2026

NEWS

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

AstraZeneca Reports FDA Approval of Saphnelo for Systemic Lupus Erythematosus (SLE)

Tanabe Pharma Report the EC Approval of Onerji for Parkinson’s Disease (PD)

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

Sanofi Reports the CHMP Positive Opinion for Cenrifki (Tolebrutinib) to Treat Non-Relapsing SPMS

Novartis’ Itvisma Receives the CHMP Positive Opinion for Spinal Muscular Atrophy

Arrowhead Pharmaceuticals Receives the CHMP’s Positive Opinion for Redemplo (Plozasiran) to Treat Familial Chylomicronemia Syndrome (FCS)

Regeneron Reports the US FDA Accelerated Approval of Otarmeni (lunsotogene parvec-cwha) in Genetic Hearing Loss

DemeRx Reports the US FDA IND Application Acceptance to Advance DMX-1001 for Alcohol Use Disorder

Regeneron and Sanofi Report the US FDA Approval of Dupixent (Dupilumab) for Chronic Spontaneous Urticaria (CSU)

Crinetics Pharmaceuticals Report the EC Approval of Palsonify for Acromegaly

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