The European Medicine Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) last week endorsed 12 new medicines for authorization, including the first treatment to prevent respiratory syncytial virus (RSV) in newborns, driving the total number of endorsed treatments to 70 since the beginning of the year. Five of the 12 approval recommendations are for orphan drugs.   The new RSV treatment, Beyfortus (nirsevimab) developed by AstraZeneca and Sanofi, was s...

The US Food and Drug Administration (FDA) on Thursday released draft guidance explaining its thought process for rescinding breakthrough therapy designation (BTD) during its evaluation of a drug.   While FDA has long asserted its authority to rescind or withdraw BTDs, the new guidance will add clarity for sponsors as to when the agency might revoke the designation. Since designation was created under the Food and Drug Administration Safety and Innovation Act in 2012,...

The US Food and Drug Administration (FDA) has authorized the first in vitro diagnostic to help diagnose early Alzheimer’s disease. The agency said the test minimizes radiation risks to patients who otherwise would need to be tested with positron emission tomography (PET) scans.   On 4 May, FDA announced it has given the green light to Fujirebio Diagnostics’ de novo application for its Lumipulse G β-Amyloid Ratio (1-42/1-40) test, which is intended for patients ages...

Developing therapies for rare diseases involves complex incentives, unique requirements, and often extensive patient engagement. The authors of RAPS’ Orphan Drug Development for Rare Diseases , Sundar Ramanan, PhD, MBA, and Sunny Kamlesh Dave, MPharm, recently took time to discuss with RAPS Senior Editor Gloria Hall the importance of orphan drugs, the peculiarities and challenges of obtaining an orphan drug designation (ODD), and what is covered in the book. During a “m...

Pediatric oncology patients continue to have significant unmet medical need. Although the Pediatric Research Equity Act (PREA) mandated evaluation of new drugs for pediatric patients, the rarity and uniquity of pediatric cancers allowed for waivers or exemptions from PREA requirements. The passing of FDA Reauthorization Act amendments in 2017 included the Research Acceleration for Cure and Equity Act, which expands the scope of oncology products subject to PREA. Sponsors d...

  A new draft guidance from the US Food and Drug Administration (FDA) gives cell and gene therapy developers a detailed framework for the conduct of umbrella trials, offering the potential for enhanced speed and efficiency in early-stage clinical trials.   In the umbrella trials envisioned under the draft guidance, two or more versions of a cellular or gene therapy product would be studied for one specific disease using just one trial design, shared infrastructure, a...

An initiative by the European Commission (EC) to boost the development of pediatric drugs and medicines for people with rare diseases has received stakeholder feedback that in many cases expresses concern with the proposed upending of the status quo.   A 60-day period of public consultation for the initiative’s roadmap closed at the end of July; in all, 112 comments were received from individuals, non-governmental organizations (NGOs), public authorities, medical socie...

A strong local presence in EU markets is needed when seeking labelling exemptions that help reduce the cost of providing orphan products, according to experts who spoke at RAPS Euro Convergence 2021.   Manufacturers of orphan drugs can gain exemptions from some labelling requirements at an EU level and from individual member states, a potentially valuable flexibility for companies that make orphan drugs targeting very small numbers of patients. Such products face speci...

In its April meeting, the European Medicines Agency’s (EMA’s) human medicines committee recommended eight new medicines for approval, including three new non-orphan medicinal products and two orphan medicines.   EMA’s Committee for Medical Products for Human Use (CHMP) also issued positive opinions for extensions of therapeutic indications for nine medicines, bringing the total new indications for 2021 to 23.   Leo Pharma’s Adtralza (tralkinumab) was recommended ...

On Monday, the US Food and Drug Administration (FDA) announced a series of efforts and a day-long conference centered on addressing the unmet needs of patients with rare diseases. FDA leaders announced the initiatives in recognition of Rare Disease Day, observed on February 28.   A new request for applications (RFA) issued by FDA augments the agency’s Orphan Products Grants Program. The RFA is seeking natural history studies to address unmet needs in rare diseases, l...

The US Food and Drug Administration (FDA) on Monday launched a new cloud-based submission portal for orphan drug designation requests, shifting away from the current paper and CD-based submission process. The new portal, first announced in January, is part of the agency’s orphan drug technology modernization effort and fits in with its broader technology modernization plans. The shift to electronic submissions follows a decade of increasing orphan drug designation reques...

Officials from the US Food and Drug Administration (FDA) provided updates on the agency’s efforts to support the development of medical products for rare diseases at the National Organization for Rare Disorders (NORD) Summit this week.   In his keynote address on Thursday, FDA Commissioner Stephen Hahn said that FDA had already approved 51 orphan indications through July of this year and acknowledged that the agency’s Office of Orphan Products Development (OOPD) has se...