Quality Systems

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Treatments for autoimmune diseases, multiple cancers, and skin blistering have been recommended for marketing approval.

EMA Recommends Four Medicines for Approval in February

Patrick Lavery is an Editor for the MJH Life Sciences brands 

. Previously at MJH, he filled the same role for 

Cerebrotendinous xanthomatosis, or CTX, is a metabolic disorder caused by a gene mutation that results in a deficiency of an important enzyme in the ability of the human body to break down fats.

FDA Approves First Treatment for Rare Lipid Storage Disease Cerebrotendinous Xanthomatosis

Seladelpar, which is marketed in the US as Livdelzi, was recommended for an orphan drug designation by CHMP in December 2024.

Gilead Treatment for PBC Granted EC Conditional Marketing Authorization

Tenosynovial giant cell tumor (TGCT) is rare and non-malignant, forming in or near joints, but which if left untreated can cause damage to joints and surrounding tissue, potentially leading to significant disability.

FDA Grants Full Approval for Symptomatic Tenosynovial Giant Cell Tumor Treatment

The agency has received certification with the Eco-Management and Audit Scheme for its commitment to environmental sustainability.

EMA Earns EMAS Certification

Feliza Mirasol is the science editor for 

The approval makes Evrysdi the first and only tablet for treating spinal muscular atrophy.

FDA Approves Evrysdi Tablet from Roche for Spinal Muscular Atrophy

With the approval, FDA granted SpringWorks a rare pediatric disease priority review voucher.

FDA Approves SpringWorks Therapeutics Treatment for Adult and Pediatric Patients with NF1-PN

Acoramidis was approved by FDA in November 2024 and is prescribed in the US under the brand name Attruby.

European Commission Approves BridgeBio Treatment for ATTR-CM

While this approval addresses breast cancer, olaparib in combination with durvalumab was approved by the EU in August 2024 for treatment of endometrial cancer.

Scottish Medicines Consortium Accepts AstraZeneca’s Breast Cancer Treatment

These infections can be caused by certain Gram-negative microorganisms, making the infections challenging to control due to high antimicrobial resistance.

Regulatory Oversight and Compliance