Clinical Research Informer

Dersimelagon phosphate by Mitsubishi Tanabe Pharma for Porphyria (Erythropoietic Protoporphyri): Likelihood of Approval

Pharmaceutical Technology

JANUARY 22, 2024

Dersimelagon phosphate is under clinical development by Mitsubishi Tanabe Pharma and currently in Phase III for Porphyria (Erythropoietic Protoporphyri).

Clinical Development

Clinical Development Development

Alnylam gets NICE backing for porphyria therapy Givlaari

pharmaphorum

OCTOBER 21, 2021

UK cost-effectiveness watchdog NICE is set to recommend NHS use of Alnylam’s gene-silencing therapy Givlaari in England and Wales for the rare disease acute hepatic porphyria (AHP), after its advisors issued a positive verdict on the drug. — British Porphyria Association (@BPA_Porphyria) October 21, 2021.

Gene Silencing

Gene Silencing Gene RNA Drugs

Report: North America is key hub for the development of porphyria therapies

BioPharma Reporter

NOVEMBER 3, 2021

The porphyria therapies market is projected to grow at an annualized rate of 10.97% during the period 2021-2030, claims a new report from Roots Analysis.

Development

Development Marketing

Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data

XTalks

OCTOBER 26, 2021

After initial rejection from the National Institute for Health and Care Excellence (NICE) last year, the non-departmental public body of the Department of Health in England has now given the green light to the gene silencing treatment Givlaari (givosiran) for the treatment of the rare metabolic disorder, acute intermittent porphyria (AIP).

Gene Silencing

Gene Silencing Gene Clinical Trials Clinical Trials

NICE recommends gene silencing therapy for porphyria patients on NHS

Pharma Times

OCTOBER 21, 2021

Givlaari uses ‘gene silencing’ RNA interference technology, to target the production of pathogenic compounds in people AHP

Gene Silencing

Gene Silencing Gene RNA Production

FDA Approves First Interchangeable Humira Biosimilar + Gene Silencing Treatment for Porphyria Gets Nod From England’s NICE – Xtalks Life Science Podcast Ep. 34

XTalks

OCTOBER 27, 2021

Ayesha also talked about a new gene silencing treatment for porphyria called Givlaari that received recommendation from England’s NICE after having been initially rejected by the health watchdog last year. Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data.

Gene Silencing

Gene Silencing Life Science FDA Approval Gene

FDA approves Alnylam’s ultra-rare disease drug Oxlumo

pharmaphorum

NOVEMBER 24, 2020

Alnylam’s Givlaari, FDA-approved a year ago for acute hepatic porphyria, costs $575,000 per year at full price in the US, although after discounts the figure is more likely to be in the region of $442,000 per year. In Europe, the company said it will reach separate pricing deals with each member state as quickly as possible.

FDA Approval

FDA Approval Gene Silencing Drugs RNA

Novo Nordisk snaps up RNAi partner Dicerna in $3.3bn takeover deal

pharmaphorum

NOVEMBER 18, 2021

Since then, Alnylam has claimed approval for two more RNAis, Givlaari (givosiran) for acute hepatic porphyria (AHP) and Oxlumo (lumasiran) for primary hyperoxaluria type 1 (PH1).

Gene Silencing

Gene Silencing RNA Protein Gene

Can The Next Generation Drug Conjugates Revolutionize The Rare Disorder Domain?

Roots Analysis

OCTOBER 13, 2023

Givlaari ® , also known as Givosiran, is a ligand mediated RNAi conjugate which utilizes N-acetyl galactosamine (GalNAc) sugar moiety as its targeting ligand and δ-aminolevulinic acid synthase 1 (ALAS1)-directed small interfering RNA for targeting the acute hepatic porphyria (AHP).

Drugs

Drugs Antibody RNA Genetics

Novo Nordisk’s gene silencing alliance with Dicerna bears first fruit

pharmaphorum

JANUARY 6, 2021

In 2018, Alnylam’s Onpattro (patisiran) became the first ever RNAi drug to be approved by the FDA, getting a green light to treat nerve damage caused by the rare disease hereditary transthyretin-mediated amyloidosis (hATTR).

Gene Silencing

Gene Silencing Gene RNA Contract Manufacturing

Emerging Research Continues To Transform Erythropoietic Protoporphyria Market Outlook

Delveinsight

FEBRUARY 22, 2021

However, it is one of a group of disorders known as porphyrias, a rare condition, Erythropoietic protoporphyria (EPP). Skin gets itchy, tingling, and turns red due to burning. Its signs and symptoms might have pointed out the existence of vampires.

Marketing

Marketing Research Cosmetics Gene

GalNAc Conjugates – Spearheading the Innovation for Conjugated Drugs

Roots Analysis

DECEMBER 18, 2023

Givlaari® (developed by Alnylam) became the first next generation GalNac conjugate to receive the FDA approval for the treatment of acute hepatic porphyria (AHP) in November 2019. Alnylam Pharmaceuticals was the first company to develop the next generation conjugate, wherein GalNAc is conjugated to aminolevulinate synthase 1 targeted si-RNA.

Drugs

Drugs FDA Approval Antibody Development

UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD

XTalks

FEBRUARY 14, 2022

Related: Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data. In October last year, NICE approved Alnylam’s Givlaari (givosiran), another gene therapy, for the treatment of the rare metabolic disorder, acute intermittent porphyria (AIP).

Gene Therapy

Gene Therapy Drugs Gene Gene Silencing

Leading innovators in miRNA chemical synthesis for the pharmaceutical industry

Pharmaceutical Technology

MARCH 9, 2023

The company’s RNAi therapies include Onpattro, which is used in the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis), Givlaari for acute hepatic porphyria, and Oxlumo for primary hyperoxaluria type 1 (PH1).

Antibody

Antibody Hormones DNA Genome

Clinical Research Informer

Dersimelagon phosphate by Mitsubishi Tanabe Pharma for Porphyria (Erythropoietic Protoporphyri): Likelihood of Approval

Alnylam gets NICE backing for porphyria therapy Givlaari

Trending Sources

Report: North America is key hub for the development of porphyria therapies

Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data

NICE recommends gene silencing therapy for porphyria patients on NHS

FDA Approves First Interchangeable Humira Biosimilar + Gene Silencing Treatment for Porphyria Gets Nod From England’s NICE – Xtalks Life Science Podcast Ep. 34

FDA approves Alnylam’s ultra-rare disease drug Oxlumo

Novo Nordisk snaps up RNAi partner Dicerna in $3.3bn takeover deal

Can The Next Generation Drug Conjugates Revolutionize The Rare Disorder Domain?

Novo Nordisk’s gene silencing alliance with Dicerna bears first fruit

Emerging Research Continues To Transform Erythropoietic Protoporphyria Market Outlook

GalNAc Conjugates – Spearheading the Innovation for Conjugated Drugs

UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD

Leading innovators in miRNA chemical synthesis for the pharmaceutical industry

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