27 June 2017 EMA/COMP/336592/2017

Inspections, Human Medicines Pharmacovigilance and Committees Division

Committee for Orphan Medicinal Products (COMP) meeting report on the review of applications for orphan designation June 2017

The Committee for Orphan Medicinal Products held its 190th plenary meeting on 13-15 June 2017.

Orphan medicinal product designation Positive opinion(s) The COMP adopted 9 positive opinions recommending the following medicines for designation as orphan medicinal products to the European Commission: 1. Opinion(s) adopted at the second COMP discussion, following the sponsor’s response to the COMP list of questions: •

Oxymetazoline hydrochloride for treatment of spinal cord injury, RDD Pharma Limited;



Recombinant human antibody directed against misfolded human superoxide dismutase 1 for treatment of amyotrophic lateral sclerosis, The Medical & Regulatory Partnership Limited;



Sirolimus for treatment of pachyonychia congenita, Raremoon Consulting Ltd.

2. Opinions adopted at the first COMP discussion: •

(S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine for treatment of gastrointestinal stromal tumours, PhaRA bvba;



Autologous CD4+ and CD8+ T cells expressing a CD19-specific chimeric antigen receptor for treatment of diffuse large B-cell lymphoma, Celgene Europe Limited;



Bacillus subtilis oxalate decarboxylase for treatment of primary hyperoxaluria, Allena Pharmaceuticals Ireland Limited;



Polyphenyl(disodium 3-O-sulfo-beta-D-glucopyranuronate)-(13)-beta-D-galactopyranoside for treatment of anti-MAG neuropathy, SFL Regulatory Affairs Consulting Ltd;

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© European Medicines Agency, 2017. Reproduction is authorised provided the source is acknowledged.



Retinol for prevention of retinopathy of prematurity, Orphanix GmbH;



Tirapazamine for treatment of hepatocellular carcinoma, PhaRA bvba.

3. Opinion(s) following appeal procedures None 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. Please also refer to the Community Register of orphan medicinal products for human use.

Negative opinion(s) The COMP did not adopt any negative opinions recommending the refusal of orphan medicinal product designations to the European Commission (EC).

Lists of questions The COMP adopted 12 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 6 oral hearings took place.

Withdrawals of applications for orphan medicinal product designation The COMP noted that 7 applications for orphan medicinal product designation were withdrawn by the sponsor before adoption of the COMP opinion.

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 granted by the European Commission since the last COMP meeting is provided in Annex 2.

Re-assessment of orphan designation at time of marketing authorisation (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 Community Register of orphan medicinal products for human use:

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/336592/2017

Page 2/8

1. Opinion(s) adopted at time of CHMP opinion: •

Oxervate (cenegermin) for treatment of neurotrophic keratitis, Dompé farmaceutici S.p.A. (EU/3/15/1586). The opinion was adopted by written procedure after the May meeting.

2. Opinion(s) following appeal procedures None Details of the designated orphan medicinal products that have been subject of a new European Union (EU) marketing authorisation application since the last COMP monthly report are provided in Annex 3. Details on the authorised orphan medicinal products can be found on the EMA website.

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

Protocol assistance advice

Upcoming meetings •

The 191th meeting of the COMP will be held on 11-13 July 2017.

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 details of 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/336592/2017

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Annex 1 Overview for orphan medicinal product designation procedure since 2000 Please also refer to the Community Register of orphan medicinal products for human use. Year

Applications submitted

Applications discussed in

Positive COMP opinions

Applications withdrawn

2

Final negative COMP opinions

EC

Orphan medicinal

designations

reporting year

4

included in authorised therapeutic indication 4

105

125

70 (56%)

54 (43%)

1 (1%)

71

7

7

2016

330

304

220 (72%)

82 (27%)

2 (1%)

209

14

14

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

119

118

86 (73%)

31 (26%)

1 (1%)

73

6

7

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

2007

3

authorised

Orphan designations

2017

2008

2

products

3

Revision of the figures for 2015, 2014, 2003, 2002, 2001 and 2000 The number of orphan medicinal products authorised includes the products for which the market exclusivity has expired. The market authorisation of an orphan medicinal product 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/265114/2017

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Year

Applications

Applications

Positive COMP

Applications

Final negative

EC

Orphan medicinal

Orphan designations

submitted

discussed in

opinions

withdrawn

COMP opinions

designations

products

included in authorised

authorised

therapeutic indication

reporting year 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

2820

2664

1897 (71%)

743 (28%)

24 (1%)

1876

135

149

Committee for Orphan Medicinal Products (COMP) meeting report on the review of applications for orphan designation EMA/COMP/265114/2017

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Annex 2 Designations granted by the European Commission following COMP opinion on the fulfilment of the orphan designation criteria since last COMP plenary meeting Please also refer to the Community Register of orphan medicinal product for human use. The list includes designation decisions that were revised following the amendment of an existing designated condition (identified by * when applicable) Active substance 225

Ac-lintuzumab

Ciclosporin*

Orphan indication

Sponsor

COMP opinion date

EC designation date

11 April 2017

22 May 2017

Treatment of acute myeloid

Voisin Consulting

leukaemia

S.A.R.L.

Treatment of bronchiolitis

PARI Pharma GmbH

11 April 2017

24 May 2017

11 April 2017

22 May 2017

11 April 2017

22 May 2017

11 April 2017

22 May 2017

obliterans syndrome Chimeric locked nucleic acid deoxynucleoside

Treatment of cutaneous T-cell

Miragen Therapeutics

phosphorothioate-linked oligonucleotide

lymphoma

Europe Ltd

Poly(oxy-1,2-ethanediyl),.alpha.-hydro-

Treatment of paroxysmal

Best Regulatory

.omega.-hydroxy-,15,15'-diester with N-acetyl-

nocturnal haemoglobinuria

Consulting Ltd

Treatment of haemophilia A

Coté Orphan Consulting

inhibitor directed against microRNA-155-5p

L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-Ltryptophyl-L-glutaminyl-L-.alpha.-aspartyl-Ltryptophylglycyl-L-alanyl-L-histidyl-L-arginyl-Lcysteinyl-L-threonyl-2-[2-(2aminoethoxy)ethoxy]acetyl-N6-carboxy-Llysinamide cyclic (2.fwdarw.12)-(disulfide); where two identical synthetic peptide domains are covalently linked at the ends of the polyethylene glycol chain Recombinant adeno-associated viral vector serotype 6 encoding the B-domain-deleted

UK Limited

Committee for Orphan Medicinal Products (COMP) meeting report on the review of applications for orphan designation EMA/COMP/265114/2017

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Active substance

Orphan indication

Sponsor

COMP opinion date

EC designation date

11 April 2017

22 May 2017

TMC Pharma Services Ltd

11 April 2017

22 May 2017

human factor VIII Recombinant human interleukin-7 fused to a

Treatment of idiopathic CD4

NeoImmuneTech, INC.,

hybrid crystallisable fragment region of a human

lymphocytopenia

Spółka Akcyjna, Oddział w Polsce

antibody Sodium (1R,3R,4R,5S)-3-({2-N-acetylamino-2-

Treatment of acute myeloid

deoxy-3-O-[(1S)-1-carboxylato-2-

leukaemia

cyclohexylethyl]-beta-D-galactopyranosyl}oxy)4-({6-deoxy-alpha-L-galactopyranosyl}oxy)-5ethyl-cyclohexan-1-yl-(38-oxo2,5,8,11,14,17,20,23,26,29,32,35-dodecaoxa39-azahentetracontan-41-yl) carboxamide Tamoxifen citrate

Treatment of cystic fibrosis

GB Pharma Srl

11 April 2017

22 May 2017

Ursodeoxycholic acid

Treatment of Niemann-Pick

IntraBio Ltd

11 April 2017

22 May 2017

disease

Committee for Orphan Medicinal Products (COMP) meeting report on the review of applications for orphan designation EMA/COMP/265114/2017

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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 last COMP monthly report Please also refer to the Community Register of orphan medicinal products for human use. Active substance

Designated orphan indication

Sponsor/applicant

EU designation number

Masitinib

Treatment of amyotrophic lateral sclerosis

AB Science

EU/3/16/1722

Viable t-cells

a)Treatment in haematopoietic stem cell transplantation

Kiadis Pharma Netherlands B.V.

EU/3/16/1678

b) Treatment of acute myeloid leukaemia

EU/3/14/1356

c) Prevention of Graft-versus-Host Disease

EU/3/08/561

Committee for Orphan Medicinal Products (COMP) meeting report on the review of applications for orphan designation EMA/COMP/265114/2017

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COMP meeting report on the review of applications for orphan ...

Jun 27, 2017 - Public summaries of opinions will be available on the EMA website following adoption of the .... where two identical synthetic peptide domains.

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