Regulatory Oversight and Compliance

FDA's approval will allow Kedrion to manufacture Ryplazim (plasminogen, human-tvmh), the only FDA-approved therapy for treating PLGD-1, at facility in Bolognana, Italy.

FDA's approval of eladocagene exuparvovec-tneq (Kebilidi) makes that product the first FDA-approved gene therapy for treating AADC deficiency.

CHMP has granted Eisai Europe and Biogen a positive opinion for the use of their monoclonal antibody therapy in treating early Alzheimer’s disease.

Balversa (erdafitinib) marks the first and only bladder cancer therapy to target FGFR3 alterations, with a demonstrated increase in overall survival from 7.8 months to 12.1 months.

CDER Director Patrizia Cavazzoni and CBER Director Peter Marks provided an update on the rare disease innovation hub in a new FDA Voices blog post.

The treatment for ATTR in certain adults with polyneuropathy had just been recommended for approval across the European Union and had gained regulatory approval in the United States at the end of 2023.

Vyalev, also known as Produodopa, has been approved in 35 countries, with more than 4200 patients having started treatment.

Pfizer’s bivalent RSV prefusion F vaccine has been approved to prevent lower respiratory tract disease resulting from the respiratory syncytial virus in adults 18 to 59 years of age who are at increased risk.

As many as 30% of people living with severe hemophilia A develop inhibitors such as TFPI, which can be produced by the body’s immune response to clotting factors in replacement therapy, often limiting the effectiveness of that therapy.

Wainzua had been previously approved for use by patients with ATTRv-PN in the United States, under the brand name Wainua.

The agency’s CHMP recommended drugs to treatments for hemophilia, two biosimilars for psoriasis, and two flu vaccines at their October meeting.

The agency has reconfirmed its recommendation to not give positive opinion to Translarna (ataluren) after a reexamination of the available data.

The draft joint network strategy for the EU until 2028 will be open for public consultation until Nov. 30, 2024.

Regulators, EMA and HMA, have published principles and recommendations on the use of LLMs, which are being increasingly used for daily tasks.

The new mechanism of action targets cholinergic receptors instead of the standard dopamine receptors.

The agency is recommending that the market authorization for Oxbryta (voxelotor) be suspended so that emerging data may be reviewed.

Two drugs, Miplyffa (arimoclomol) and Aqneursa (levacetylleucine), have been approved by FDA to treat neurological symptoms associated with Niemann-Pick disease, type C in adults and children.

FDA’s Darby Kozak provided commentary on the anniversary of the Drug Price Competition and Patent Term Restoration Act of 1984.

The approval of EBGLYSS (lebrikizumab-lbkz) by FDA puts a new biologic treatment for moderate-to-severe atopic dermatitis on the market for patients whose symptoms are not well controlled with topical treatments.

Approvals were recommended for treatments of ovarian cancer, lung cancer, bleeding disorders, respiratory disease, and more.

Bio/pharma companies should be approaching their decarbonization journey as a strategic business move for the future as industry stakeholders are placing greater value on sustainability.

Phase III trial results showed that Imfinzi (durvalumab in combination with chemotherapy reduced the risk of recurrence, progression, or death by 32% in patients with resectable non-small cell lung cancer before and after surgery.

PiaSky (crovalimab) has previously been approved in countries such as the United States and Japan for monthly, subcutaneous use that may be self-administered with adequate training.

Opaganib was given orphan drug designation for the treatment of neuroblastoma in children, which is rare, but the most common infancy malignancy.

In a 24-week study of patients with PAH, six-minute walking distance improved by an average of 40.8 meters, and risk of death or clinical worsening was reduced by 82%.

The combination of Opdivo plus Yervoy is already established as a second-line treatment for patients with advanced hepatocellular carcinoma.

The International Coalition of Medicines Regulatory Authorities’ new Collaborative Hybrid Inspection Pilot program allows a CDMO to gain approval from multiple countries simultaneously, as opposed to separately phased inspections from different corners of the globe.

Fasenra, which has been approved since 2019 in the United States in a pre-filled, single-use auto-injector form that can be administered either in a doctor’s office or at home, is now approved in China for age 12 and up.

Approximately 15% of all lung cancers diagnosed in the US are of the small-cell variety, and of those, 70% are considered extensive-stage.

NexoBrid, a topically administered biological product, is now authorized for use in all age groups in the United States, European Union, and Japan.