European Union: European Medicines Agency's - Human Medicines: Highlights Of 2017

On January 23, 2018, the European Medicines Agency (EMA) has published its annual report titled 'Human medicines: Highlights of 201741. In the report, the EMA has published an overview of its key recommendations of 2017 regarding the authorization of new medicines and the safety monitoring of medicines.

Advances in medicines authorizations are essential for public health as they have the potential to improve treatment of diseases. In 2017, EMA recommended 92 medicines for marketing authorization. Of these, 35 had a new active substance, which has never been authorized in the European Union (EU) before. Many of these medicines represent a significant improvement in their therapeutic areas; they include medicines for children, for rare diseases and advanced therapies42. Amongst the 35 new active substances (NAS) that EMA recommended, 11 were new drugs and biologics to treat cancer, 05 to treat neurological disorders, 04 for infectious diseases, 04 for immunology/rheumatology, 03 for endocrinology, 02 each for Uroephrology, haematology, and dermatology, 01 for Pneumonology, and 01 for hepatology/gastroenterology class of drugs.

Table 1: List of New Active Substances Approved by the European Medicines Agency

Sr. No. Medicine Approved Therapeutic Area
1. Axumin (Fluciclovine) Cancer/Oncology
2. Bavencio (Avelumab) Cancer/Oncology
3. Besponsa (inotuzumab ozogamicin) Cancer/Oncology
4. Fotivda (Tivozanib) Cancer/Oncology
5. Kisqali (Ribociclib) Cancer/Oncology
6. Lutathera (Lutetium (177Lu) oxodotreotide) Cancer/Oncology
7. Rydapt (Midostaurin) Cancer/Oncology
8. Tecentriq (Atezolizumab) Cancer/Oncology
9. Tookad (Padeliporfin) Cancer/Oncology
10. Varuby (Rolapitant) Cancer/Oncology
11. Zejula (Niraparib) Cancer/Oncology
12. Brineura (Cerliponase alfa) Neurology
13. Ocrevus (Ocrelizumab) Neurology
14. Oxervate (Recombinant human Nerve Growth factor (rhNGF)) Neurology
15. Reagila (Cariprazine hydrochloride) Neurology
16. Spinraza (Nusinersen) Neurology
17. Maviret (Glecaprevir/pibrentasvir) Infections
18. Prevymis (Letermovir) Infections
19. Trumenba (Meningococcal group B vaccine (recombinant, adsorbed)) Infections
20. Vosevi (Sofosbuvir / Velpatasvir / Voxilaprevi) Infections
21. Kevzara (Sarilumab) Immunology/Rheumatology
22. Spherox (Spheroids of human autologous matrix-associated chondrocytes) Immunology/Rheumatology
23. Tremfya (Guselkumab) Immunology/Rheumatology
24. Xeljanz (Tofacitinib) Immunology/Rheumatology
25. Crysvita (Burosumab) Endocrinology
26. Ozempic (Semaglutide) Endocrinology
27. Xermelo (Telotristat etiprate) Endocrinology
28. Lokelma (Sodium zirconium cyclosilicate) Uro-nephrology
29. Veltassa (Patiromer sorbitex calcium) Uro-nephrology
30. Adynovi (Rurioctocog alfa pegol) Haematology
31. Refixia (Nonacog beta pegol) Haematology
32. Dupixent (Dupilumab) Dermatology
33. Kyntheum (Brodalumab) Dermatology
34. Alofisel (Expanded human allogeneic mesenchymal adult stem cells extracted from adipose tissue) Hepatology/Gastroenterology
35. Fasenra (Benralizumab) Penumology

EMA's Special Program Approvals

EMA, in its report, also highlighted its expedited programs and incentives to bring products to market more quickly and spur development of products to treat rare diseases or other areas of high unmet medical need.

Orphan Medicines

To qualify for agency's orphan designation, a medicine must meet a number of criteria43:

  • It must be intended for treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;
  • The prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;
  • No satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorized, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.

In 2017, the agency approved 19 drugs in its Orphan Medicine Program, out of which 13 were New Active Substances.

Accelerated Assessment

Accelerated assessment reduces the timeframe for the EMA's Committee for Medicinal Products for Human Use (CHMP) to review a marketing authorization application. Applications may be eligible for accelerated assessment if the CHMP decides that the product is of major interest for public health and therapeutic innovation44. This mechanism is reserved for medicines that are able to address unmet medical needs. It allows for faster assessment of eligible medicines by EMA's scientific committees (within up to 150 days rather than up to 210 days).

In 2017, seven medicines received a recommendation for marketing authorization following an accelerated assessment out of which five were new active substances.

Conditional marketing authorizations

The EMA supports development of medicines that address unmet medical needs of patients. In the interest of public health, applicants may be granted a conditional marketing authorization for such medicines where the benefit of immediate availability outweighs the risk of less comprehensive data than normally required, based on the scope and criteria defined in relevant legislation and guidelines45.

In 2017, three medicines received a recommendation for conditional marketing authorization out of which two were new active substances.

Approval under exceptional circumstances

Approval under exceptional circumstances is an agency's regulatory pathway that allows patients access to medicines that cannot be approved under a standard authorization as comprehensive data cannot be obtained, either because there are only very few patients with the disease, or the collection of complete information on the efficacy and safety of the medicine would be unethical, or there are gaps in the scientific knowledge. These medicines are subject to specific post-authorization obligations and monitoring46.

In 2017, two medicines were authorized under exceptional circumstances.

Table 2: Number of Drugs Approved by EMA Special Program Approvals

Program or Designation New Active Substances Approved Total Approvals
Orphan Medicine 13 19
Accelerated Assessment 05 07
Conditional Marketing Authorization 02 03
Approval Under Exceptional Circumstances 01 02

New uses for existing medicines

51 extensions of indication were recommended in 2017. The extension of the use of a medicine that is already approved in a new therapeutic indication can also offer new treatment opportunities for patients.

EMA's Safety Monitoring

Once a medicine is placed on the market, EMA and the EU Member States continuously monitor the quality and the benefit/risk balance of the medicine under its authorized conditions of use. In 2017, EMA gave new safety advice to manage risks observed with a number of medicines on the market in the EU. Regulatory measures ranged from a change in the product information to the suspension or withdrawal of a medicine to even recall of a limited number of batches.

Important new safety advice issued in 2017 included:

  • Information about a potential increased risk of lower limb amputation (mostly affecting the toes) in patients taking the SGLT2 inhibitors canagliflozin, dapagliflozin and empagliflozin used for type 2 diabetes.
  • Recommendations to restrict the use of some linear gadolinium agents used in MRI body scans and suspend the authorizations of others. EMA's scientific review found that small amounts of gadolinium may remain in the brain after a scan with these agents, although there is currently no evidence that these small amounts cause any harm.
  • Recommendation to restrict the use of Zinbryta for treating multiple sclerosis, in view of the risk of serious liver damage in some patients.
  • Recommendation to suspend marketing of paracetamol medicines designed to release the active ingredient over a long period (modified-release medicines) because of the difficulty in managing overdoses.
  • New recommendation on medicines containing a combination of dienogest 2 mg and ethinylestradiol 0.03 mg which can continue to be used to treat moderate acne when certain other treatments have failed, but should only be used in women who also choose oral contraception.
  • Prescription information for the antibiotic vancomycin to be changed to ensure appropriate use in the treatment of serious infections caused by Gram-positive bacteria. The recommendation aims to ensure appropriate use in the context of the fight against antimicrobial resistance.
  • New contraindication for Uptravi, which must not be taken simultaneously with medicines like gemfibrozil which are strong blockers (inhibitors) of the liver enzyme CYP2C8.
  • New recommendation for Symbioflor 2 to continue to be used to treat irritable bowel syndrome (IBS), but not for other functional gastrointestinal disorders. The company will provide a study on effectiveness and safety among patients with different features of IBS.
  • eview of human factor VIII medicines authoriszd in the EU - EMA concluded that there is no clear evidence of a difference in the risk of inhibitor development between the two classes of factor VIII medicines. Patients should therefore continue to use their factor VIII medicines as prescribed by the doctor.
  • ew contraception recommendations for male patients regarding concerns that mycophenolate medicines (used to prevent rejection of transplanted organs) cause miscarriages or birth defects.
  • New recommendation on injectable methylprednisolone medicines (used to treat the symptoms of severe allergic reactions) containing lactose which must not be used in patients with a known or suspected allergy to the proteins in cow's milk.

Conclusion:

In 2017, the EMA saw an increase in the number of new/novel drugs and biologics it recommended for marketing authorization. New Drugs/ Novel drugs are innovative products that serve previously unmet medical needs or otherwise significantly help to advance patient care and public health.

Footnotes

41. http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2018/01/news_detail_002886.jsp&mid=WC0b01ac058004d5c1

42. http://www.ema.europa.eu/docs/en_GB/document_library/Report/2018/01/WC500242079.pdf

43. http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000029.jsp

44. http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000955.jsp&mid=WC0b01ac05809f843a

45. http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000925.jsp

46. http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000925.jsp

The content of this article is intended to provide a general guide to the subject matter. Specialist advice should be sought about your specific circumstances.

To print this article, all you need is to be registered on Mondaq.com.

Click to Login as an existing user or Register so you can print this article.

Authors
Similar Articles
Relevancy Powered by MondaqAI
 
In association with
Related Topics
 
Similar Articles
Relevancy Powered by MondaqAI
Related Articles
 
Related Video
Up-coming Events Search
Tools
Print
Font Size:
Translation
Channels
Mondaq on Twitter
 
Register for Access and our Free Biweekly Alert for
This service is completely free. Access 250,000 archived articles from 100+ countries and get a personalised email twice a week covering developments (and yes, our lawyers like to think you’ve read our Disclaimer).
 
Email Address
Company Name
Password
Confirm Password
Position
Mondaq Topics -- Select your Interests
 Accounting
 Anti-trust
 Commercial
 Compliance
 Consumer
 Criminal
 Employment
 Energy
 Environment
 Family
 Finance
 Government
 Healthcare
 Immigration
 Insolvency
 Insurance
 International
 IP
 Law Performance
 Law Practice
 Litigation
 Media & IT
 Privacy
 Real Estate
 Strategy
 Tax
 Technology
 Transport
 Wealth Mgt
Regions
Africa
Asia
Asia Pacific
Australasia
Canada
Caribbean
Europe
European Union
Latin America
Middle East
U.K.
United States
Worldwide Updates
Registration (you must scroll down to set your data preferences)

Mondaq Ltd requires you to register and provide information that personally identifies you, including your content preferences, for three primary purposes (full details of Mondaq’s use of your personal data can be found in our Privacy and Cookies Notice):

  • To allow you to personalize the Mondaq websites you are visiting to show content ("Content") relevant to your interests.
  • To enable features such as password reminder, news alerts, email a colleague, and linking from Mondaq (and its affiliate sites) to your website.
  • To produce demographic feedback for our content providers ("Contributors") who contribute Content for free for your use.

Mondaq hopes that our registered users will support us in maintaining our free to view business model by consenting to our use of your personal data as described below.

Mondaq has a "free to view" business model. Our services are paid for by Contributors in exchange for Mondaq providing them with access to information about who accesses their content. Once personal data is transferred to our Contributors they become a data controller of this personal data. They use it to measure the response that their articles are receiving, as a form of market research. They may also use it to provide Mondaq users with information about their products and services.

Details of each Contributor to which your personal data will be transferred is clearly stated within the Content that you access. For full details of how this Contributor will use your personal data, you should review the Contributor’s own Privacy Notice.

Please indicate your preference below:

Yes, I am happy to support Mondaq in maintaining its free to view business model by agreeing to allow Mondaq to share my personal data with Contributors whose Content I access
No, I do not want Mondaq to share my personal data with Contributors

Also please let us know whether you are happy to receive communications promoting products and services offered by Mondaq:

Yes, I am happy to received promotional communications from Mondaq
No, please do not send me promotional communications from Mondaq
Terms & Conditions

Mondaq.com (the Website) is owned and managed by Mondaq Ltd (Mondaq). Mondaq grants you a non-exclusive, revocable licence to access the Website and associated services, such as the Mondaq News Alerts (Services), subject to and in consideration of your compliance with the following terms and conditions of use (Terms). Your use of the Website and/or Services constitutes your agreement to the Terms. Mondaq may terminate your use of the Website and Services if you are in breach of these Terms or if Mondaq decides to terminate the licence granted hereunder for any reason whatsoever.

Use of www.mondaq.com

To Use Mondaq.com you must be: eighteen (18) years old or over; legally capable of entering into binding contracts; and not in any way prohibited by the applicable law to enter into these Terms in the jurisdiction which you are currently located.

You may use the Website as an unregistered user, however, you are required to register as a user if you wish to read the full text of the Content or to receive the Services.

You may not modify, publish, transmit, transfer or sell, reproduce, create derivative works from, distribute, perform, link, display, or in any way exploit any of the Content, in whole or in part, except as expressly permitted in these Terms or with the prior written consent of Mondaq. You may not use electronic or other means to extract details or information from the Content. Nor shall you extract information about users or Contributors in order to offer them any services or products.

In your use of the Website and/or Services you shall: comply with all applicable laws, regulations, directives and legislations which apply to your Use of the Website and/or Services in whatever country you are physically located including without limitation any and all consumer law, export control laws and regulations; provide to us true, correct and accurate information and promptly inform us in the event that any information that you have provided to us changes or becomes inaccurate; notify Mondaq immediately of any circumstances where you have reason to believe that any Intellectual Property Rights or any other rights of any third party may have been infringed; co-operate with reasonable security or other checks or requests for information made by Mondaq from time to time; and at all times be fully liable for the breach of any of these Terms by a third party using your login details to access the Website and/or Services

however, you shall not: do anything likely to impair, interfere with or damage or cause harm or distress to any persons, or the network; do anything that will infringe any Intellectual Property Rights or other rights of Mondaq or any third party; or use the Website, Services and/or Content otherwise than in accordance with these Terms; use any trade marks or service marks of Mondaq or the Contributors, or do anything which may be seen to take unfair advantage of the reputation and goodwill of Mondaq or the Contributors, or the Website, Services and/or Content.

Mondaq reserves the right, in its sole discretion, to take any action that it deems necessary and appropriate in the event it considers that there is a breach or threatened breach of the Terms.

Mondaq’s Rights and Obligations

Unless otherwise expressly set out to the contrary, nothing in these Terms shall serve to transfer from Mondaq to you, any Intellectual Property Rights owned by and/or licensed to Mondaq and all rights, title and interest in and to such Intellectual Property Rights will remain exclusively with Mondaq and/or its licensors.

Mondaq shall use its reasonable endeavours to make the Website and Services available to you at all times, but we cannot guarantee an uninterrupted and fault free service.

Mondaq reserves the right to make changes to the services and/or the Website or part thereof, from time to time, and we may add, remove, modify and/or vary any elements of features and functionalities of the Website or the services.

Mondaq also reserves the right from time to time to monitor your Use of the Website and/or services.

Disclaimer

The Content is general information only. It is not intended to constitute legal advice or seek to be the complete and comprehensive statement of the law, nor is it intended to address your specific requirements or provide advice on which reliance should be placed. Mondaq and/or its Contributors and other suppliers make no representations about the suitability of the information contained in the Content for any purpose. All Content provided "as is" without warranty of any kind. Mondaq and/or its Contributors and other suppliers hereby exclude and disclaim all representations, warranties or guarantees with regard to the Content, including all implied warranties and conditions of merchantability, fitness for a particular purpose, title and non-infringement. To the maximum extent permitted by law, Mondaq expressly excludes all representations, warranties, obligations, and liabilities arising out of or in connection with all Content. In no event shall Mondaq and/or its respective suppliers be liable for any special, indirect or consequential damages or any damages whatsoever resulting from loss of use, data or profits, whether in an action of contract, negligence or other tortious action, arising out of or in connection with the use of the Content or performance of Mondaq’s Services.

General

Mondaq may alter or amend these Terms by amending them on the Website. By continuing to Use the Services and/or the Website after such amendment, you will be deemed to have accepted any amendment to these Terms.

These Terms shall be governed by and construed in accordance with the laws of England and Wales and you irrevocably submit to the exclusive jurisdiction of the courts of England and Wales to settle any dispute which may arise out of or in connection with these Terms. If you live outside the United Kingdom, English law shall apply only to the extent that English law shall not deprive you of any legal protection accorded in accordance with the law of the place where you are habitually resident ("Local Law"). In the event English law deprives you of any legal protection which is accorded to you under Local Law, then these terms shall be governed by Local Law and any dispute or claim arising out of or in connection with these Terms shall be subject to the non-exclusive jurisdiction of the courts where you are habitually resident.

You may print and keep a copy of these Terms, which form the entire agreement between you and Mondaq and supersede any other communications or advertising in respect of the Service and/or the Website.

No delay in exercising or non-exercise by you and/or Mondaq of any of its rights under or in connection with these Terms shall operate as a waiver or release of each of your or Mondaq’s right. Rather, any such waiver or release must be specifically granted in writing signed by the party granting it.

If any part of these Terms is held unenforceable, that part shall be enforced to the maximum extent permissible so as to give effect to the intent of the parties, and the Terms shall continue in full force and effect.

Mondaq shall not incur any liability to you on account of any loss or damage resulting from any delay or failure to perform all or any part of these Terms if such delay or failure is caused, in whole or in part, by events, occurrences, or causes beyond the control of Mondaq. Such events, occurrences or causes will include, without limitation, acts of God, strikes, lockouts, server and network failure, riots, acts of war, earthquakes, fire and explosions.

By clicking Register you state you have read and agree to our Terms and Conditions