Regulatory Filings and Submissions

RoslinCT will manufacture Vertex Pharmaceuticals’ CRISPR-based gene therapy, Casgevy, recently approved for treating sickle cell disease and β thalassemia.

Exagamglogene autotemcel seen as synechdoche.

Recommended medicines include treatments for generalized myasthenia gravis, glioma, neovascular age-related macular degeneration, and more.

The designation will grant Lisata EU marketing exclusivity for 10 years after approval and protocol assistance from EMA.

The recommended medicines include treatments for Duchenne muscular dystrophy, relapsed or refractory multiple myeloma, hyperargininemia, invasive candidiasis, menopause symptoms, and diagnostic medicines for characterizing lesions.

The agency recommended conditional marketing authorization for Elrexfio (elranatamab) as a monotherapy for adult patients with relapsed and refractory multiple myeloma.

The draft guidance document discusses quality considerations for topical ophthalmic drug products.

Janssen has requested an extension to the indication application of its fully-human, bispecific antibody treatment, Rybrevant (amivantamab).

Eli Lilly and Company has received a complete response letter from FDA for its anti-dermatitis biologic therapeutic, lebrikizumab.

The European Commission has granted approval for Tyruko (natalizumab), a biosimilar developed by Polpharma Biologics for treating multiple sclerosis.

The agency will be reorganizing field force and compliance functions.

Otsuka Pharmaceutical Europe and Astex Pharmaceuticals have announced the approval of INAQOVI by the European Commission.

As for the topic of subjectivity, Viehmann said how high levels of subjectivity in risk assessments and in QRM are problematic and are not aligned with the 1st QRM principle of Q9.

The EMA’s Committee for Medicinal Products for Human Use has given a positive opinion on Sandoz’ biosimilar trastuzumab for breast and gastric cancer.

The draft guidance document discusses the development of labeling for proposed biosimilars and interchangeable biosimilars for submission under section 351(k) of the Public Health Service Act.

The safety profile of erdafitinib observed in THOR was consistent with the previously reported safety profile of erdafitinib in metastatic urothelial carcinoma (mUC).

The updated vaccine will be ready to ship following a positive European Commission review.

FDA has approved a new mAb therapy from Regeneron Pharmaceuticals as well as a higher dose version of Eylea, the company’s eye disease therapeutic.

SBS-518 is a first-in-class dual sigma receptor (SR) antagonist/dopamine transporter (DAT) inhibitor in development for the treatment of stimulant use disorder (StUD).

The investigational drug was developed to activate a novel uptake pathway, allowing antineoplastic drugs to combat solid tumors more successfully.

The nasal-spray drug can be used to reverse the effects of opioid overdose.

What impact will EMA’s decision to phase out COVID-19 regulatory flexibilities have on the pharma industry?

The new drug may help vulnerable children resist RSV in the coming fall and winter season.

EMA looks to focus on mRNA vaccines because their classification depends on the target and/or whether they are obtained chemically or biologically.

FDA's approval of Leqembi paves the way for wider coverage of the drug by Medicare and establishes a process for further clinical testing and evaluation of treatments for this widespread, debilitating condition

FDA has approved Roctavian (valoctocogene roxaparvovec-rvox), a gene therapy from BioMarin Pharmaceutical for treating severe hemophilia A in adults.

Preparation is essential for regulatory meetings—it not only crystallizes what is needed from the regulators, it helps them better understand the development programme and potential challenges.

Changes to PRIME scheme are set to drive greater harmonization across major pharmaceutical markets.

The agency approved Elevidys to treat pediatric patients four through five years of age with Duchenne muscular dystrophy.

An increase in applications for gene therapies is putting stress on FDA’s resources.