Regulatory Filings and Submissions

FDA expects more than 200 investigational new drug applications for cell and gene therapies by 2020, causing the agency to strengthen its regulatory program.

The agency clarified the process for development programs for regenerative medicine therapies.

The guidance explains the process of applying for competitive generic therapy designation.

A taskforce evaluated the advantage of using Big Data in the evaluation of the benefits versus risks of medications.

The drug is approved in the United States specifically for treating acquired thrombotic thrombocytopenic purpura, a rare blood-clotting disorder.

In a statement, FDA Commissioner Scott Gottlieb announced the agency’s plans to publish guidance to promote the development of complex generic drugs.

FDA staffers will be hard-pressed to process and review the wave of new drug application submissions in pre-established timeframes for action.

Full commercial launch of the drug is underway in the United States, with Europe to follow.

Cell and gene therapy platform company, Locate Bio, has announced that it is set to receive key patents for its IntraStem technology covering the United States and Europe

The agency revised draft guidance to enable more efficient and successful drug development programs for the treatment or prevention of rare diseases.

Sanofi’s Adacel (tetanus toxoid, reduced diphtheria toxoid and acellular pertussis [Tdap] vaccine adsorbed) is approved for a repeat dose in people 10 through 64 years of age, 8 years or more after the first vaccination.

The revised guidance aims to strengthen the agency’s global approach to develop new antibacterial medicines.

The plan details five goals to guide the development of Sentinel, a national electronic system for medical product safety surveillance established as part of the FDA Amendments Act of 2007, over the next five years.

In 2018, the agency authorized 42 drugs with new active substances, three advanced therapies, and 21 orphan drugs.

FDA plans to support initiatives to ensure that all medicines are safe, effective, and of high quality.

The agency sent a warning letter to Roche’s Genetech for marketing unapproved stem cell products and puts other stem cell firms and providers on notice.

FDA has approved Truxima (rituximab-abbs), a biosimilar to Roche’s anti-cancer biologic, Rituxan (rituximab).

Shire has announced that the European Commission has granted marketing authorisation for Takhzyro (lanadelumab) subcutaneous injection.

The positive opinions included the first oral-only tablet for the treatment of human African trypanosomiasis.

The agency provided an update on its relocation plans and assured that core activities are continuing uninterrupted.

The new good pharmacovigilance practice chapter IV on specific considerations for the pediatric population offers a holistic view of pediatric pharmacovigilance and provides guidance on how to address the specific needs and challenges of safety monitoring of medicines used in children.

The decision follows an FDA request for additional information to complement the company’s submission for biosimilar rituximab.

The company received FDA approval for Hyrimoz (adalimumab-adaz), its biosimilar referencing AbbVie’s blockbuster Humira (adalimumab).

Mylan announced the launch of its biosimilar to AbbVie’s Humira across major European markets.

The companies announced the European launch of Imraldi (adalimumab), a biosimilar referencing AbbVie’s blockbuster Humira (adalimumab).

Takhzyro (lanadelumab) is the first monoclonal antibody therapy approved for the prevention of recurrent attacks of hereditary angioedema (HAE) in patients aged 12 years and older.

The recommended drugs include one biosimilar, two orphan medicines, and three extensions of therapeutic indication.

To achieve a more dynamic marketplace, FDA is issuingguidance documents and targeted advisories to support R&D on complex generics and combination products.

The guidance describes the agency's recommendations on how to group patients with different molecular alterations and approaches for evaluating the benefits and risks of targeted therapies where some molecular alterations may occur at low frequencies.

The agency is developing technology- and disease-specific regulatory frameworks for innovations that may not have previously had a clear development pathway.