26 May 2016 EMA/COMP/331689/2016

Procedure Management and Committees Support Division

Committee for Orphan Medicinal Products (COMP) meeting report on the review of applications for orphan designation May 2016 The Committee for Orphan Medicinal Products held its 178th plenary meeting on 17-19 May 2016.

Orphan medicinal product designation Positive opinions The COMP adopted 19 positive opinions recommending the following medicines for designation as orphan medicinal products to the European Commission (EC): 1. Opinions adopted at the second COMP discussion, following the sponsor’s response to the COMP list of questions: •

2'-O-(2-methoxyethyl) phosphorothioate antisense oligonucleotide targeting the growth hormone receptor for treatment of acromegaly, Coté Orphan Consulting UK Limited;



3-(5-amino-2-methyl-4-oxoquinazolin-3(4H)-yl)piperidine-2,6-dione hydrochloride for treatment of diffuse large B-cell lymphoma, Celgene Europe Limited;



Allogeneic donor-derived ex-vivo expanded T lymphocytes transduced with a retroviral vector containing inducible caspase 9 and truncated CD19 for treatment in haematopoietic stem cell transplantation, QRC Consultants Ltd;



Citric acid monohydrate for treatment of acute liver failure, CATS Consultants GmbH;



Donor T lymphocytes depleted ex vivo of host alloreactive T cells using photodynamic treatment for treatment in haematopoietic stem cell transplantation, Kiadis Pharma Netherlands B.V.;

• •

Eflornithine for treatment of glioma, Orbus Therapeutics Limited; Humanised anti-IL-6 receptor monoclonal antibody for treatment of neuromyelitis optica spectrum disorders, Chugai Pharma Europe Ltd;



Humanised monoclonal antibody targeting interleukin-15 for treatment of eosinophilic oesophagitis, Dr Alain Vicari;

30 Churchill Place ● Canary Wharf ● London E14 5EU ● United Kingdom Telephone +44 (0)20 3660 6000 Facsimile +44 (0)20 3660 5555 Send a question via our website www.ema.europa.eu/contact

An agency of the European Union

© European Medicines Agency, 2016. Reproduction is authorised provided the source is acknowledged.



Melatonin for treatment of neonatal sepsis, Therapicon Srl;



Melatonin for treatment of necrotising enterocolitis, Therapicon Srl;



Molgramostim for treatment of acute respiratory distress syndrome, Serendex Pharmaceuticals A/S.

2. Opinions adopted at the first COMP discussion: •

Cyclocreatine for treatment of creatine deficiency syndromes, Pharma Gateway AB;



Diclofenamide for treatment of periodic paralysis, Sun Pharmaceutical Industries Europe B.V.;



Modified mRNA encoding the UGT1A1 protein for treatment of Crigler-Najjar syndrome, Alexion Europe SAS;



Pyridoxine and L-pyroglutamic acid for treatment of fragile X syndrome, FGK Representative Service Ltd.;



Recombinant humanised monoclonal IgG2 lambda antibody against human sclerostin for treatment of osteogenesis imperfecta, Mereo Biopharma Group Limited;



Recombinant protein derived from the saliva of the Ornithodoros moubata tick for treatment of Guillain-Barré syndrome, Akari Therapeutics Plc;



Setmelanotide for treatment of Prader-Willi syndrome, TMC Pharma Services Ltd;



Teriparatide for treatment of hypoparathyroidism, Alacrita LLP.

Public summaries of opinions will be available on the EMA website following adoption of the respective decisions on orphan designation 1 by the European Commission.

Lists of questions The COMP adopted 10 lists of questions on initial applications. These applications will be discussed again at the next COMP meeting prior to the adoption of an opinion.

Oral hearings 11 oral hearings took place.

Withdrawals of applications for orphan medicinal product designation The COMP noted that 1 application for orphan medicinal product designation was withdrawn.

Detailed information on the orphan designation procedures An overview of orphan designation procedures since 2000 is provided in Annex 1. The list of medicinal products for which decisions on orphan designation have been given by the European Commission since the last COMP meeting is provided in Annex 2.

1

Details of all orphan designations granted to date by the European Commission are entered in the EU Register of Orphan Medicinal Products Committee for Orphan Medicinal Products (COMP) meeting report on the review of applications for orphan designation EMA/COMP/331689/2016

Page 2/8

Applications for marketing authorisation for orphan medicinal products Details of those designated orphan medicinal products that have been subject of a new European Union (EU) marketing authorisation application through the centralised procedure since the last COMP plenary meeting are provided in Annex 3. Details on the authorised orphan medicinal products can be found on the EMA website.

Article 5(12) (b) of Regulation (EC) No 141/2000 of the European Parliament and of the Council In line with its responsibility to review whether or not a designated orphan medicinal product still fulfils the designation criteria prior to the granting of a marketing authorisation, the COMP adopted 1 opinion recommending to the European Commission that the following orphan medicinal product be kept in the EU registry of orphan medicinal product: •

Gazyvaro (obinutuzumab) for treatment of follicular lymphoma, Roche Registration Limited (EU/3/15/1504).

Other matters The main topics addressed during the meeting related to: •

Protocol assistance advice

Upcoming meetings •

The 179th meeting of the COMP will be held on 14-16 June 2016.

Note This monthly report, together with other information on the work of the European Medicines Agency, can be found on the EMA website: www.ema.europa.eu Contact our press officer Monika Benstetter Tel. +44 (0)20 3660 8427 E-mail: [email protected]

Committee for Orphan Medicinal Products (COMP) meeting report on the review of applications for orphan designation EMA/COMP/331689/2016

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Annex 1 Overview for orphan medicinal product designation procedure since 2000 Year

Applications submitted

Applications discussed in

Positive COMP opinions

Applications withdrawn

2

Final negative COMP opinions

EC

Orphan medicinal

designations

reporting year

2 3

products

3

authorised

Orphan designations included in authorised therapeutic indication

2016

98

106

82 (77%)

24 (23%)

0

58

4

4

2015

258

272

177 (65%)

94 (35%)

1 (1%)

190

14

21

2014

329

259

196 (76%)

62 (24%)

2 (1%)

187

15

16

2013

201

197

136 (69%)

60 (30%)

1 (1%)

136

7

8

2012

197

192

139 (72%)

52 (27%)

1 (1%)

148

10

12

2011

166

158

111 (70%)

45 (29%)

2 (1%)

107

5

5

2010

174

176

123 (70%)

51 (29%)

2 (1%)

128

4

4

2009

164

136

113 (83%)

23 (17%)

0 (0%)

106

9

9

2008

119

118

86 (73%)

31 (26%)

1 (1%)

73

6

7

2007

125

117

97 (83%)

19 (16%)

1 (1%)

98

13

13

2006

104

103

81 (79%)

20 (19%)

2 (2%)

80

9

11

2005

118

118

88 (75%)

30 (25%)

0 (0%)

88

4

4

2004

108

101

75 (74%)

22 (22%)

4 (4%)

73

6

6

2003

87

96

54 (56%)

37 (40%)

1 (1%)

55

5

5

2002

80

75

43 (57%)

32 (42%)

2 (3%)

49

4

4

2001

83

90

62 (70%)

26 (29%)

1 (1%)

64

3

3

2000

72

32

26 (81%)

3 (10%)

0 (0%)

14

0

0

Total

2483

2341

1689 (72%)

631 (27%)

21 (1%)

1654

118

132

Revision of the figures for 2015, 2014, 2003, 2002, 2001 and 2000 Number of authorised orphan medicinal products may cover more than one orphan designation

Committee for Orphan Medicinal Products (COMP) meeting report on the review of applications for orphan designation EMA/COMP/331689/2016

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Annex 2 Medicinal products granted a European Union designation as orphan medicinal product by the European Commission since the April 2016 COMP monthly report Active substance

Orphan indication

Sponsor

COMP opinion date

EC designation date

(1E,6E)-1,7-bis(3,4-dimethoxyphenyl)-4-

Treatment of X-linked spinal and

Coté Orphan Consulting

23 March 2016

28 April 2016

cyclobutylmethyl-1,6-heptadiene-3,5-dione

bulbar muscular atrophy

UK Limited Celgene Europe Limited

23 March 2016

28 April 2016

ProQR Therapeutics BV

23 March 2016

28 April 2016

Treatment of epidermolysis bullosa

Intrexon Actobiotics N.V.

23 March 2016

28 April 2016

Treatment of systemic sclerosis

Cytori Ltd

23 March 2016

28 April 2016

Prevention of cytomegalovirus

Chimerix UK Ltd

23 March 2016

28 April 2016

Prevention of graft-versus-host

Richardson Associates

23 March 2016

28 April 2016

disease

Regulatory Affairs Ltd

Combination of 4-hydroxyandrostenedione,

Treatment of multiple symmetric

Dr Regenold GmbH

23 March 2016

28 April 2016

Serenoa serrulata fruit extract and alpha lipoic

lipomatosis

Development·Regulatory· 23 March 2016

28 April 2016

(Kennedy’s disease) 2-methyl-1-[(4-[6-(trifluoromethyl)pyridin-2-

Treatment of acute myeloid

yl]-6-{[2-(trifluoromethyl)pyridin-4-yl]amino}-

leukaemia

1,3,5-triazin-2-yl)amino]propan-2-ol methanesulfonate Antisense oligonucleotide complementary to the

Treatment of Leber’s congenital

exonic splicer enhancer sequence atintron 26 of

amaurosis

the centrosomal protein 290 pre-mRNA Autologous dermal fibroblasts genetically modified ex vivo with a lentiviral vector containing the human COL7A1 gene Autologous stromal vascular cell fraction from adipose tissue Brincidofovir

disease Cannabidiol

acid Fluocinolone acetonide

Market Access Treatment of non-infectious uveitis

Campharm Ltd

Committee for Orphan Medicinal Products (COMP) meeting report on the review of applications for orphan designation EMA/COMP/331689/2016

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Active substance

Orphan indication

Sponsor

COMP opinion date

EC designation date

Humanised recombinant IgG4 anti-human tau

Treatment of progressive

Abbvie Ltd

23 March 2016

28 April 2016

antibody

supranuclear palsy

Human/murine chimeric monoclonal antibody

Treatment of soft tissue sarcoma

Tracon Pharma Limited

23 March 2016

28 April 2016

N-carboxymethyl-glycyl-L-threonyl-L-histidyl-L-

Treatment of beta thalassaemia

QRC Consultants Ltd

23 March 2016

28 April 2016

3,3-diphenylalanyl-L-piperidincarboxy-3-yl-L-

intermedia and major

Voisin Consulting

23 March 2016

28 April 2016

Herantis Pharma Plc

23 March 2016

28 April 2016

Galderma R&D

23 March 2016

28 April 2016

Acies Bio d.o.o.

23 March 2016

28 April 2016

23 March 2016

28 April 2016

23 March 2016

28 April 2016

against endoglin

arginyl-L-S-methylthio-cystyl-L-arginyl-Ltryptophyl-aminohexanyl-Ncarboxamidomethyl-glycine N-hexadecylamide Recombinant adeno-associated viral vector

Treatment of Angelman syndrome

serotype 9 carrying the gene for the human E6-

S.A.R.L.

AP ubiquitin protein ligase Recombinant human cerebral dopamine

Treatment of amyotrophic lateral

neurotrophic factor

sclerosis

Resiquimod

Treatment of cutaneous T-cell lymphoma

S-acetyl-(S)-4'-phosphopantetheine, calcium

Treatment of pantothenate-

salt

kinase-associated neurodegeneration

Tyr-Met-Phe-Pro-Asn-Ala-Pro-Tyr-Leu, Ser-Gly-

Treatment of acute myeloid

SELLAS Life Sciences

Gln-Ala-Tyr-Met-Phe-Pro-Asn-Ala-Pro-Tyr-Leu-

leukaemia

Group UK, Limited

Tyr-Met-Phe-Pro-Asn-Ala-Pro-Tyr-Leu, Ser-Gly-

Treatment of malignant

SELLAS Life Sciences

Gln-Ala-Tyr-Met-Phe-Pro-Asn-Ala-Pro-Tyr-Leu-

mesothelioma

Group UK, Limited

Pro-Ser-Cys-Leu-Glu-Ser, Arg-Ser-Asp-Glu-LeuVal-Arg-His-His-Asn-Met-His-Gln-Arg-Asn-MetThr-Lys-Leu and Pro-Gly-Cys-Asn-Lys-Arg-TyrPhe-Lys-Leu-Ser-His-Leu-Gln-Met-His-Ser-ArgLys-His-Thr-Gly

Pro-Ser-Cys-Leu-Glu-Ser, Arg-Ser-Asp-Glu-LeuCommittee for Orphan Medicinal Products (COMP) meeting report on the review of applications for orphan designation EMA/COMP/331689/2016

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Active substance

Orphan indication

Sponsor

COMP opinion date

EC designation date

Val-Arg-His-His-Asn-Met-His-Gln-Arg-Asn-MetThr-Lys-Leu and Pro-Gly-Cys-Asn-Lys-Arg-TyrPhe-Lys-Leu-Ser-His-Leu-Gln-Met-His-Ser-ArgLys-His-Thr-Gly

Committee for Orphan Medicinal Products (COMP) meeting report on the review of applications for orphan designation EMA/COMP/331689/2016

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Annex 3 Designated orphan medicinal products that have been subject of a new European Union marketing authorisation application under the centralised procedure since the April 2016 COMP monthly report Active substance

Designated orphan indication

Sponsor/applicant

EU designation number

Pacritinib

a) Treatment of post-essential

Baxalta Innovations GmbH

EU/3/10/767

thrombocythemia myelofibrosis b) Treatment of post-polycythemia vera

EU/3/10/769

myelofibrosis c) Treatment of primary myelofibrosis Tivozanib hydrochloride monohydrate

Treatment of renal cell carcinoma

EU/3/10/768 EUSA PHARMA

Committee for Orphan Medicinal Products (COMP) meeting report on the review of applications for orphan designation EMA/COMP/331689/2016

EU/3/10/747

Page 8/8

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