EMA | Leaders in Pharmaceutical Business Intelligence (LPBI) Group

Posts Tagged ‘EMA’

Real Time Coverage @BIOConvention #BIO2019: Genome Editing and Regulatory Harmonization: Progress and Challenges

Posted in and Bioethics, Biotechnology, Conference Coverage with Social Media, CRISPR applied to Human Germ Line, CRISPR/Cas9 & Gene Editing, FDA, FDA Regulatory Affairs, FDA, CE Mark & Global Regulatory Affairs: process management and strategic planning - GCP, GLP, ISO 14155, Gene Editing Impact on Longevity, Gene Therapy & Gene Editing Development, Health Law & Patient Safety, Health Law Policy, REAL TIME Conference Coverage Twitter's Hashtags and Handles per Presentation/session, tagged biosafety and bioethics, CRISPR, CRISPR Biology • Genome Editing • Gene Drives • DNA Repair • Model and Industrial Organisms • Technology Development • Stem Cells/Cancer • Genome-Wide Screens, Duchenne muscular dystrophy, EMA, EUDRA, European Union, FDA, FDA 510(k) clearance, gene editing on June 5, 2019| Leave a Comment »

Real Time Coverage @BIOConvention #BIO2019: Genome Editing and Regulatory Harmonization: Progress and Challenges

Reporter: Stephen J Williams, PhD @StephenJWillia2

Genome editing offers the potential of new and effective treatments for genetic diseases. As companies work to develop these treatments, regulators are focused on ensuring that any such products meet applicable safety and efficacy requirements. This panel will discuss how European Union and United States regulators are approaching therapeutic use of genome editing, issues in harmonization between these two – and other – jurisdictions, challenges faced by industry as regulatory positions evolve, and steps that organizations and companies can take to facilitate approval and continued efforts at harmonization.

Speakers

Emily MardenCounsel Food Drug and Regulatory Life Sciences at Sidley Austin LLP

Gary CharbonneauSenior Vice President of Regulatory Affairs at Sarepta

Dr. Denise GavinActing Gene Therapy Branch Chief, Division of Cell and Gene Therapy at Center for Biologics Evaluation and Research (CBER), FDA

Georgia GavriilidouCounsel, Food, Drug and Medical Device Regulatory Life Sciences at Sidley Austin LLP

Daniel TakefmanSVP and Head of Regulatory Affairs at Spark Therapeutics

CBER: because of the nature of these gene therapies, which are mainly orphan, there is expedited review. Since they started this division in 2015, they have received over 1500 applications.

Spark: Most of the issues were issues with the primary disease not the gene therapy so they had to make new endpoint tests so had talks with FDA before they entered phase III. There has been great collaboration with FDA, now they partnered with Novartis to get approval outside US. You should be willing to partner with EU pharmas to expedite the regulatory process outside US. In China the process is new and Brazil is behind on their gene therapy guidance. However there is the new issue of repeat testing of your manufacturing process, as manufacturing of gene therapies had been small scale before. However he notes that problems with expedited review is tough because you don’t have alot of time to get data together. They were lucky that they had already done a randomized trial.

Sidley Austin: EU regulatory you make application with advance therapy you don’t have a national option, the regulation body assesses a committee to see if has applicability. Then it goes to a safety committee. EU has been quicker to approve these advance therapies. Twenty five percent of their applications are gene therapies. Companies having issues with manufacturing. There can be issues when the final application is formalized after discussions as problems may arise between discussions, preliminary applications, and final applications.

Sarepta: They have a robust gene therapy program. Their lead is a therapy for DMD (Duchenne’s Muscular Dystrophy) where affected males die by 25. Japan and EU have different regulatory applications and although they are similar and data can be transferred there is more paperwork required by EU. The US uses an IND for application. Global feedback is very challenging, they have had multiple meetings around the world and takes a long time preparing a briefing package….. putting a strain on the small biotechs. No company wants to be either just EU centric or US centric they just want to get out to market as fast as possible.

Please follow LIVE on TWITTER using the following @ handles and # hashtags:

@Handles

@pharma_BI

@AVIVA1950

@BIOConvention

# Hashtags

#BIO2019 (official meeting hashtag)

#itstartswithone

#RightMixMatters

Read Full Post »

Future of Calcitonin…?

Posted in Bone Disease and Musculoskeletal Disease, CANCER BIOLOGY & Innovations in Cancer Therapy, tagged Committee for Medicinal Products for Human Use, EMA, European Medicines Agency, European Union, Food and Drug Administration, osteoporosis, United States, Upsher-Smith Laboratories on July 20, 2012| 16 Comments »

Reported by: Dr. V.S. Karra, Ph.D.

The European equivalent of the US Food and Drug Administration (FDA) yesterday recommended withdrawing calcitonin nasal spray — indicated for treating osteoporosis in the European Union — because of an increased risk for cancer.
The European Medicines Agency (EMA) also said that the long-term use of calcitonin-containing medicines delivered by injection or infusion increases the risk for cancer. Calcitonin in any formulation should not be used to treat osteoporosis at all, the agency said.

In the United States, 2 nasal-spray versions of calcitonin are FDA-approved for treating postmenopausal osteoporosis in women: Fortical (Upsher-Smith Laboratories) and Miacalcin (Novartis). Neither of the labels for the 2 drugs contains restrictions on how they should be used or a warning about the risk for cancer.

Calcitonin, also called calcitonin-salmon, is a synthetic copy of a polypeptide hormone secreted by the ultimobranchial gland of salmon.

The EMA said it based its recommendations on a review of the benefits and risks of calcitonin-containing medicines. Conducted by the agency’s Committee for Medicinal Products for Human Use (CHMP), the review encompassed available data from the companies that market these drugs, postmarketing safety data, randomized controlled studies, 2 studies of unlicensed oral calcitonin drugs, and experimental cancer studies, among other sources.

CHMP found that “a higher proportion of patients treated with calcitonin for long periods of time develop cancer of various types, compared with patients taking placebo.” The increase in cancer rates ranged from 0.7% for oral formulations to 2.4% for the nasal formulation. CHMP concluded that the benefits of calcitonin for osteoporosis did not exceed the risks. The nasal spray’s only indication is for osteoporosis, thus justifying the drug’s removal from the market.

As a solution for injection or infusion, calcitonin should be administered for no more than 4 weeks to prevent acute bone loss resulting from sudden immobilization, and normally for no more than 3 months to treat Paget’s disease, the EMA said. The agency did not specify a time frame for the short-term use of calcitonin for treating hypercalcemia caused by cancer.

For further details go to their website

Source