Regulatory Filings and Submissions

Stelara (ustekinumab), already approved for treating adults, is now also approved for treating adolescents with moderate to severe plaque psoriasis.

The committee has voted unanimously to approve Spark Therapeutics’ gene therapy candidate, Luxturna (voretigene neparvovec), for treating a genetically inherited blindness.

Amgen is seeking approval for an additional indication in glucocorticoid-induced osteoporosis for its blockbuster osteoporosis therapeutic, Prolia.

AstraZeneca is seeking approval for its anti-cancer monoclonal antibody, Imfinzi, for treating non-small cell lung cancer in the European Union.

The approval marks the first biosimilar approved in the United States for treating cancers.

FDA issued a Refusal to File letter to Acorda Therapeutics, citing insufficiently complete information in the company’s new drug application for its investigational Parkinson’s disease drug.

The withdrawal has been attributed to issues at the biotech company’s manufacturing facility.

FDA urges manufacturers to seek fast approval of “high-need” generics and targeted therapies. 

The agency is offering patients with life-threatening diseases a how-to tool on accessing investigational drugs.

The agency announced a plan to eliminate its existing orphan designation request backlog.

Approval of breakthrough therapies requires expedited quality assessment.

CBER is moving forward with the development and approval of regenerative medicine advanced therapies.

Sandoz, a Novartis division, announced  that the Committee for Medicinal Products for Human Use (CHMP) has adopted positive opinions recommending the approval of its biosimilars rituximab and  etanercept in Europe, for the same indications as the respective reference medicines.

The agency approved Renflexis, a biosimilar to Janssen’s blockbuster rheumatoid arthritis treatment.

Pharmaceutical Technology spoke with CPhI North America presenter Jonathan Helfgott to discuss navigating GDUFA and helpful tips for submitting successful ANDAs.

The mAb is the first approved treatment that targets the progressive form of the disease.

Will new generic drugs bring the cost of medicines down in the way policy makers hope?

In a FDAVoice blog post, CBER Director Peter Marks discusses the new designation for cell therapies that treat life-threatening diseases.

Moving global manufacturing operations may be more complicated than it appears.

In 2016, FDA approved 630 ANDAs and tentatively approved 183 ANDAs, the highest number to date, according to the report.

FDA granted inotuzumab ozogamicin priority review and accepted its BLA for filing.

The regulatory agency rejected the medication, citing various issues related to device use.

FDA approved Valeant’s brodalumab with a boxed warning for suicidal ideation.

Drug companies are pushing Congress to action on funding for FDA programs and staff to expedite drug reviews and approvals.

A new report from HBM Partners said FDA new molecular entity approvals have dropped to 19 in 2016 from 45 in 2015.

FDA’s Center for Drug Evaluation and Research (CDER) approved 22 new molecular entities (NMEs) in 2016, according to a January 4, 2016 release from the agency. Of the 22 NMEs, 12 were large-molecule therapeutics. Of CDER’s 45 novel drug approvals in 2015, 17 were considered large-molecule therapeutics (larger than 900 Daltons).In 2016, 11 of the 12 large-molecule drugs that were approved were biologics. This includes seven monoclonal antibodies (mAbs), one hormone, and three DNA-derived medications. The remaining large-molecule medication was a diagnostic agent.

The agency recommended nine drugs including treatments for diabetes, hepatitis B, and HIV.

What’s ahead for the healthcare and pharmaceutical industries?

NICE recommended eribulin for the treatment of patients with breast cancer, reversing its 2012 decision.

FDA leaders insist that the decision to approve Sarepta’s Exondys (eteplirsen) should not be considered a model for future development of orphan drugs.