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Europe’s April 2015 Products Recommended For Orphan Drug Designation


The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting April 14 – 16, 2015.

At this meeting, there are 13 positive opinions recommending the following medicines for designation as orphan medicinal products. COMP’s opinions are forwarded to the European Commission (EC). The EC will then decide whether to grant an orphan designation for the medicines in question. Public summaries of the opinions will be available on the EMA website following adoption of the respective decisions on orphan designation by the EC.

EMA COMP April 2015 ODDs Recommended

Product Name Sponsor Company Indication
{2-amino-8-[4-(pyrrolidinylcarbonyl)phenyl]-(3H-benzo[f]azepin-4-yl)}-N,N-dipropylcarboxamide Right Track Regulatory Limited Ovarian Cancer
AASSGVSTPGSAGHDIITEQPRS (P42) Centre National de la Recherche Scientifique (CNRS) Huntington’s Disease
Adeno-associated viral vector serotype 9 containing the human HGSNAT gene Cochamo Systems Ltd Mucopolysaccharidosis IIIC (Sanfilippo C Syndrome)
Fusion proteins composed by a genetically modified cholera toxin subunit A1, peptides from the acetylcholine receptor alpha chain & a dimer of the D fragment from Staphylococcus aureus protein A Toleranzia AB Myasthenia Gravis
Reduced oxydised N-acetyl heparin Sigma-Tau Pharma Ltd Plasma Cell Myeloma
Triamcinolone acetonide S-cubed Limited Non-Infectious uveitis
2-(7-ethoxy-4-(3-fluorophenyl)-1-oxophthalazin-2(1H)-yl)-N-methyl-N-(2-methylbenzo[d]oxazol-6-yl)acetamide Clinical Network Services (UK) Ltd Cystic Fibrosis
5,7-dichloro-2-dimethylaminomethyl-8-hydroxyquinoline hydrochloride Prana Biotechnology UK Limited Huntington’s Disease
Adult human bone-marrow-derived, ex-vivo-expanded, pooled allogeneic mesenchymal stromal cells Regulatory Resources Group Ltd Thromboangiitis obliterans (Buerger’s Disease)
Allopurinol sodium ACE Pharmaceuticals BV Perinatal asphyxia
Humanised anti-CD37 monoclonal antibody conjugated to maytansinoid DM1 ImmunoGen Europe Limited Diffuse large B-cell lymphoma
Trehalose Dr. Ulrich Granzer Oculopharyngeal Muscular Dystrophy
Triheptanoin Pharma Gateway AB Glucose transporter type-1 deficiency syndrome


Please Note: “Erlenmeyer Flasks” From Argonne US National Lab  [Public domain in the US] | Wikimedia Commons.

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

Orphan Drug Industry: Top 10 Inspirational Figures In Asia

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General lack of understanding of rare diseases, insufficient funding mechanisms and options, absence of legislative frameworks are but a few reasons the Asian orphan drugs market has seen minimal advancements. A sustainable access program through improved healthcare policies to protect the rare disease patients, and also to incentivize orphan drug developers, will help put an end to the market’s stagnant phase.

A list of 10 super heroes behind the scenes who’ve been pushing for more patient rights protection, healthcare policy revision, and public awareness of rare diseases in their individual state or country in Asia have been compiled in an E-Book. We introduce to you 10 inspirational figures who will further encourage industry peers to push for greater access of orphan drugs in Asia, and better coverage of drugs pricing and health insurance.

The E-Book can be downloaded here

Several of these inspirational figures will be joining us at the World Orphan Drug Congress Asia, June 3-4, 2015, in Singapore.

Meeting Logo courtesy of Terrapinn. The Orphan Druganaut Blog is a Media Partner.

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

Findacure: Launch Of Online Portal For Patient Groups

Findacure Online Portal

The Orphan Druganaut Blog is honored to have again, Flóra Raffai, the Head of Development from Findacure, as a Guest Blogger. Findacure is a charity which focuses around fundamental diseases – rare, often genetic, diseases from which the basic mechanisms of dysfunction can be extrapolated and applied to our understanding of more common conditions.

There is a chronic lack of support for rare diseases. In a 2013 study by Shire, 62% of patients stated they needed to provide their healthcare professionals with information on their disease.[1] As a result, patients turn instead of patient groups to provide much needed information. Patient groups also act as the first port of call for pharmaceutical companies and academics preparing research to develop treatments and clinical trials.

However, out of the 7000 known rare diseases, at least half do not have a disease-specific patient group. Where patient groups do exist, they usually come in the form of ‘kitchen-table’ organisations, set up by people living with rare conditions or those who have an affected family member. They transfer their skills with no previous experience in the third sector or in healthcare. There is little help available for these people, and they are themselves in need of a support group.

As a key focus of Findacure, we aim to meet that need through training programmes for rare disease groups. Today we are launching our latest project to achieve this aim: an online information portal. This portal is directed at patients and advocates looking to acquire the skills and tools they need to run their patient group.

We have worked closely with volunteers from established patient communities, international rare disease advocacy organisations, and academic institutions to develop credible and accurate toolkits for new patient groups. These toolkits come in the form of a written guide providing an overview, focusing on topics such as setting up a patient group, how to work with academics, and how to fundraise successfully. These are supplemented by collated resources which users are directed to for further information. Furthermore, each guide has a dedicated forum where patients and advocates can share their own experiences and knowledge, contributing to the collective knowledge of the rare disease community.

The portal currently contains five such guides, free to access for all patients and advocates. We are continually working on developing new guides and new content to further support users. Follow this link to sign up now.


1 Shire. 2013. Rare Disease Impact Report: Insight from patients and the medical community. Accessed Online 8th April 2015.

Image courtesy of Findacure.

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

FDA Breakthrough Therapy: Pfizer’s Xalkori For ROS1+ NSCLC


Pfizer announces April 21st, that the FDA grants the Breakthrough Therapy Designation (BTD) to Xalkori (Crizotinib). The BTD is for ROS1-positive (ROS1+) Non-Small Cell Lung Cancer (NSCLC). ROS1+ NSCLC is a subgroup of NSCLC, occurring in about 1% of NSCLC cases. Currently, Xalkori is FDA approved for metastatic NSCLC, where tumors are Anaplastic Lymphoma Kinase positive (ALK+) as determined by a FDA-approved test. Xalkori has received approval in more than 80 countries.

According to the Pfizer Press Release, Dr. Mace Rothenbery, Senior Vice President of Clinical Development and Medical Affairs, and Chief Medical Officer for Pfizer Oncology”

“Xalkori pioneered precision medicine for ALK-positive metastatic NSCLC, and ROS1 represents a second molecular subgroup of NSCLC in which Xalkori has demonstrated a level of anti-tumor activity that can potentially make a real difference for patients.”

This is the 3rd BTD Pfizer has received, with the FDA approval of 2 of the BTDs:

•   Ibrance (Palbociclib) for breast cancer in April 2013 with FDA approval in February 2015

•   Trumenba (Bivalent rLP2086 Vaccine) for Meningococcal B Disease with FDA approval in October 2014

•   Xalkori (Crizotinib) for Ros1+ NSCLC.



FDA BTD Approval Chart

FDA BTD Statistics Chart.

Please Note: “White Pocket Reflection” by John Fowler (Flickr: White Pocket Reflection) [CC BY 2.0] | Wikimedia Commons.

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

Rare Disease Gene Therapy: Milo Technology’s 1st DMD Patient In Follistatin Trial


Milo Biotechnology is a clinical stage startup company developing treatments to strengthen muscle for neuromuscular diseases. The company is developing a muscle-strengthening Follistatin protein gene therapy for Duchenne Muscular Dystrophy (DMD), in collaboration with Nationwide Children’s Hospital (Cleveland).

Milo Biotechnology announces April 9th, that the first DMD patient has been treated in a Follistatin gene therapy trial at Nationwide Children’s Hospital. The therapy is delivered by intramuscular injection. This is a 6 patient clinical trial.

Milo Biotechnology’s main investigational product is AAV1-FS344, which leads to the local expression of Follistatin, a potent TGFb ligand inhibitor inhibitor. The gene therapy technology was developed at and is exclusively licensed from Nationwide Children’s Hospital. AAV1-FS344 is an adeno-associated gene therapy. It receives FDA Orphan Drug Designation (ODD) in November 2012.

Milo Technology’s FDA ODD Database Record For DMD Gene Therapy

Generic Name: Adeno-associated virus transgene of follistatin
Trade Name: n/a
Date Designated: 11-19-2012
Orphan Designation: Treatment of Duchennes and Becker’s muscular dystrophy
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: Milo Biotechnology 2322 Delaware Drive Cleveland Heights, OH 44106


Please Note: “DNA Repair” courtesy of Tom Ellenberger, Washington University School of Medicine in St. Louis. [Public domain] | Wikimedia Commons

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

FDA Breakthrough Therapy Designation: Statistics Chart Updated (as of 04/03/15)

FDA logo



The CBER BTD #s are current as of 03/31/15 and the CDER BTD #s are current as of 04/03/15.

The changes are as follows:

•   The total # of FDA CDER + CBER BTD Requests received by the FDA increases from 293 to 295

•   The total # of FDA CDER + CBER BTDs Granted by the FDA increases from 82 to 84

•   The total # of FDA CDER + CBER BTDs Denied by the FDA increases from 155 to 58

•   The total # of FDA CDER + CBER BTDs Pending by the FDA decreases from 56 to 53.

FDA CBER BTDs + CDER BTDs (as of 04/03/15)

Breakthrough Therapy  Designation (BTD) Category Total # of CBER Designations (07/09/12-03/31/15) Total # of CDER Designations (07/09/12-04/03/15) Total # of CBER +   CDER BTD Designations (07/09/12-04/03/15)
Total # of BTD Requests Received 49 246 295
Total # of BTDs Granted 11 73 84
Total # of BTDs   Denied 34 124 158
Total # of BTDs   Pending 4 49 53



•   28.5 % of the total # of BTD Requests Received results in the BTD being granted

•   53.5 % of the total # of BTD Requests Received results in the BTD being denied

•   18.0 % of the total # of BTD Requests Received results in the BTD pending.



FDA BTD Approval Chart

FDA BTD Statistics Chart.

Please Note: FDA Official Logo from FDA website.

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

9 New FDA Orphan Drug Designations: Week of 04/13/15






The chart below identifies 9 new FDA Products Receiving Orphan Designation for the week of 04/13/15 as of 04/17/15 in ascending “Orphan Drug Designation Date” order.

FDA Products Receiving Orphan Drug Designation (Week of 04/13/15)

# Generic Name/ODD Date Sponsor Company Indication
1 Zoledronate D,L-lysine monohydrate (ZLM)/ 04.15 Thar Pharmaceuticals Complex Regional Pain Syndrome (CRPS)
2 Selumetinib/ 04.15 AstraZeneca Pharmaceuticals Uveal Melanoma
3 Obinutuzumab/ 04.15 Genentech Follicular Lymphoma
4 Triheptanoin/ 04.15 Ultragenyx Pharmaceutical Fatty Acid Oxidation Disorders
5 Pelareorep/ 04.15 Oncolytics Biotech (Canada) Malignant Glioma
6 Diphenylcyclopropenone gel/ 04.15 RXi Pharmaceuticals Malignant melanoma stage IIB to IV
7 2-[4-[[(2-R)-2-ethoxy-3-[4-(trifluoromethyl)phenoxy]propyl]thio]-2-methylphenoxy]acetic acid (1:1) lysine dihydrate/ 04.15 Cymabay Therapeutics Frederickson Type I or V hyperlipoproteinemia
8 [α−N-(2’succinyl-paclitaxel)Thr]-Phe-Phe-Tyr-Gly-Gly-Ser-Arg-Gly-[ε−N-(2’succinyl-paclitaxel)Lys]-Arg-Asn-Asn-Phe-[ε−N-(2’succinyl-paclitaxel)Lys]-Thr-Glu-Glu-Tyr / 04.15 Angiochem Brain Metastases
9 177-LU-DOTA-GlyGlyNle-CycMSHhex/ 04.16 SolaranRx Stage IIB through IV malignant melanoma


** Generic Name/ODD Date” Column Link = Is the FDA Orphan Drug Product Designation Database Record.

Please Note : “Two small test tubes held in spring clamps” courtesy of  Amitchell125 at English Wikipedia [CC-BY-SA-30] | Wikimedia Commons.

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.


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