Hospital.Help

Active ingredient: Pritelivir 100 mg Tablets Treatment of acyclovir-resistant mucocutaneous HSV (herpes simplex virus) infections in immunocompromised adults. 02.11.2023 02.11.2024 Responsible person:
AiCuris Anti-infective Cures GmbH
Friedrich-Ebert-Strasse 475
42117 Wuppertal

Contact details:
myTomorrows
Anthony Fokkerweg, 61;
1059 CP Amsterdam
Netherlands
Phone: + 31 88 525 3 888
Email: regulatory@mytomorrows.com

Active ingredient: Arimoclomol
Capsules Treatment of Niemann-Pick disease Type C in Germany 12.12.2023 12.12.2024 Responsible person:
Zevra Denmark A/S
Ole Maaløes Vej 3
DK-2200 Copenhagen
Denmark

Contact details:
Telephone:+45 39 17 82 72
E-mail:
medicalaffairs@zevra.com

Active ingredient: LOXO-292, 20,40,80 mg,
Hard capsule; 5 Gpowder for oral suspension A Multi-Center Expanded Access Program (EAP) for the Treatment of Patients with Locally Advanced or Metastatic Solid Tumors with Rearranged During Transfection (RET) Activation (LIBRETTO-201) 21.11.2023 21.11.2024

Responsible person:
Loxo Oncology Inc
281 Tresser Boulevard
CT 06901 Stamford
United States

European representative:
UBC BioSource Corporation p.L.
Avenida del Doctor Arce 14
28002 Madrid

Contact details:
Phone: +44 (0) 208 834 0100
Email: EURegulatory@ubc.com

TAS-120 (futibatinib),
4 mgfilm-coated tablet AT OPEN-LABEL COMPASSIONATE USE PROGRAM OF FUTIBATINIB (TAS-120) IN PATIENTS WITH ADVANCED CHOLANGIOCARCINOMA HARBORING FGFR2 GENE REARRANGEMENTS 18.12.2023 18.12.2024

Responsible Person:
Taiho Oncology Inc. USA

Legal Representative:
Taiho Pharma Netherlands BV,
Barbara Strozzilaan 201,
1083HN, Amsterdam, Netherlands

Contact person:
Dr. Volker Wacheck
Email: vwacheck@taihooncology.com

Leniolisib 10 mg30 mg and 70 mg Film-coated tablets Patients with activated PI3 kinase delta syndrome (APDS). 06.06.2023 06.06.2024

Responsible Person:
WEP Clinical Ireland Ltd.

Legal Representative:
WEP Clinical Ireland Ltd, 46 Upper Mount Street, Dublin 2, D02 RX88 Ireland,

Contact person:
Antoine Doyen
Email: regulatory@wepclinical.com

Tofersen (BIIB067)
6.7 mg/ml solution for injection Treatment of patients with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene 12.12.2023 12.12.2024

Responsible Person:
Biogen Inc.
225 Binney Street
Cambridge, MA 02142, USA

European Representative:
Biogen GmbH
Riedenburger Strasse 7
81677 Munich

Contact person:
Dr. Christiane Sommer
Email: christiane.sommer@biogen.com
Phone: +49 89 99617-394
Fax: +49 89 99617-199

Nedosiran (DCR-PHXC) FOR PATIENTS WITH PRIMARY HYPEROXALURIA TYPE 1 (PH1) 19.07.2023 19.07.2024

Responsible Person:
Dicerna Pharmaceuticals Inc
75 Hayden Ave
Lexington, Massachusetts 02421
UNITED STATES

Legal Representative:
German Hyperoxaluria Center
Pediatric Kidney Center & Hyperoxaluria Center
In Mühlenbach 2b
53127 Bonn

contact details:
Prof. Dr. Bernd Hoppe
Email: bernd.hoppe@knz-bonn.de

Brensocatib (INS1007)
Dosage form:
(as Monohydrate), 10mg, tablets for the treatment of non-cystic fibrosis bronchiectasis (patients completing the ASPEN trial). 18.03.2024 18.03.2025

Responsible Person:
Insmed Incorporated
700 US Highway 202/206
08807-1704 Bridgewater
UNITED STATES

Legal Representative:
Insmed Netherlands BV
City Plateau 7
3521 Utrecht, Netherlands
NETHERLANDS

Contact details:
info-benelux@insmed.com

Medicine: Iptacopan (LNP023)
Active ingredient: LNP023 hydrochloride
Dosage form: hard capsules Managed Access Program (MAP) CLNP023B12004M to provide access to iptacopan (LNP023) for Complement 3 Glomerulopathy in native and transplanted kidneys 15.04.2024 15.04.2025

Responsible Person:
Novartis Pharma GmbH;
Roonstrasse 25
90429 Nuremberg

Contact:
Novartis Pharma GmbH;
Medical information service;
Telephone: 0911-273 12100;
Fax: 0911-273 12160
Email: infoservice.novartis@novartis.com

Active ingredient: Ganaxolone
50 mg/ml, oral suspension Compassionate Use of Ganaxolone for Treatment of Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder (CDD) 06.10.2023 06.10.2024

Responsible Person:
Marinus Pharmaceuticals Emerald Limited (“Marinus”) Ireland

EU Representatives:
WEP Clinical Portugal

Contact details:
WEP Clinical Portugal,
Beloura Business Centre
100 m from the beach,
Block 4, 1 º C, Line,
2710-297 Sintra,
Portugal,
Tel +44 208 004 8190,
Fax + 44 872 111 5766
jkhera@wepclinical.com

Active ingredient: Olorofim Treatment of invasive fungal infections 27.12.2023 27.12.2024

Responsible Person:
F2G Ltd.
Lankro Way
M30 OLX Eccles, Manchester
UNITED KINGDOM

Contact details:
Emma Harvey
Emma.harvey@f2g.com

Active ingredient: Nirogacestat Treatment of progressive desmoid tumors/aggressive fibromatosis (DT/AF) 15.03.2024 15.03.2025

Responsible Person:
SpringWorks Therapeutics
100 Washington Blvd
Stamford, CT 06902

Contact details:
Email: medinfo@springworkstx.com

Active ingredient: Zilucoplan Compassionate Use Program: Zilucoplan for Participants with generalized Myasthenia Gravis (gMG) 12.07.2023 12.07.2024

Responsible Person:
UCB Pharma GmbH
Alfred-Nobel-Strasse 10
40789 Monheim

Contact details:
Email: karl-werner.leffers@ucb.com
Phone:+49-2173-48-1445

Active ingredient: Sparsentan Compassionate Use Program (CUP) with Sparsentan for the Treatment of
Primary Immunoglobulin A Nephropathy (IgAN) 08.08.2023 08.08.2024

Responsible Person:
Vifor Pharma Germany GmbH
Baierbrunner Str. 29,
81379 Munich
Germany

Contact details:
Email: ralf.nickel@viforpharma.com
Telephone: +49 172 518 1531

Active ingredient: Pirtobrutinib
100 mg Film-coated tablets For the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) who have been previously treated with a Bruton tyrosine kinase (BTK) inhibitor. 19.10.2023 19.10.2024

Responsible Person
Lilly Germany GmbH
Werner-Reimers-Strasse 2-4
61352 Bad Homburg

Contact details:
iOMEDICO
Ellen-Gottlieb-Str. 19
79106 Fribourg
cup-pirtobrutinib@iomedico.com
Phone: +49 (0)761 15 242 31

Active ingredient: Pirtobrutinib
100 mg Film-coated tablets For the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) who had previously been treated with a Bruton tyrosine kinase (BTK) inhibitor. 19.10.2023 19.10.2024

Responsible Person
Lilly Germany GmbH
Werner-Reimers-Strasse 2-4
61352 Bad Homburg

Contact details:
iOMEDICO
Ellen-Gottlieb-Str. 19
79106 Fribourg
cup-pirtobrutinib@iomedico.com
Phone: +49 (0)761 15 242 31

Active ingredient: Erdafitinib Treatment of patients with an alteration of the fibroblast growth factor receptor (FGFR) in advanced or metastatic urothelial carcinoma 15.12.2023 15.12.2024

Responsible Person:
Janssen Pharmaceutica NV
Turnhoutseweg 30
2340 Beerse
Belgium

Contact details:
Dr. Franziska Dillner
Johnson & Johnson 1st place
41470 Neuss
Phone: +49 2137 9553077

Active ingredient: Repotrectinib (BMS-986472) BMS-986472 (repotrectinib) as a treatment for adult patients with locally advanced or metastatic R0S1-positive non-small cell lung cancer 24.01.2024 24.01.2025

Responsible Person:
Bristol-Myers Squibb GmbH & Co. KGaA
Arnulfstrasse 29
80636 Munich

Contact details:
Dr. Dennis Russkamp
Manager Medical Advisor Oncology
dennis.russkamp@bms.com

Judith Ruf
Senior Trial Manager
judith.ruf@bms.com

Active ingredient: Fruquintinib (TAK-113) Fruquintinib (TAK-113) intended to treat adults with refractory metastatic colorectal cancer (mCRC) who cannot be adequately treated with current standard therapy 30.01.2024 30.01.2025

Responsible Person:
Takeda GmbH
78467 Constance
Byk-Gulden-Str. 2
78467 Constance

Contact details:
Stefan Joenleit
Phone: +49(0) 16090512604
Email: stefan.joneleit@takeda.com

Active ingredient: Pridopidine Pridopidine compassionate use programme in patients with Huntington’s Disease” (PL101-HD502) 24.01.2024 24.01.2025

Responsible Person:
Prilenia Therapeutics BV
Gooimeer 235
1411 DC Naarden
Netherlands

Contact details:
medinfo@prilenia.com

Active ingredient: Elamipretide For the treatment of patients with genetically confirmed rare diseases with known mitochondrial dysfunction 08.02.2024 08.02.2025

Responsible Person:
Stealth BioTherapeutics Inc.

Contact details:
WEP Clinical Ireland Ltd
Tel +44 208004 8190,
Fax + 44 872 111 5766.
Antoine Doyen Regulatory@wepclinical.com

Active ingredient: Emblaveo® (Avibactam-Na, Aztreonam)

Treatment of infections due to aerobic gram-negative pathogens in adults with limited treatment options

22.02.2024 22.02.2025

Responsible Person:
Pfizer Inc.
66 Hudson Boulevard East
New York, NY 10001-2192
United States of America

contact details:
Pfizer Pharmaceuticals GmbH – Berlin
Caroline Schweizer
caroline.schweizer@pfizer.com

Active ingredient: Leriglitazone Adult patients and patients aged ≥ 2 years with cerebral X-linked adrenoleukodystrophy (X-ALD). 26.02.2024 26.02.2025

Responsible Person:
Minoryx Therapeuticals SL
Av. Ernest Lluch 32, TCM3
08302 Mataro (Barcelona)

Contact details:
amistry@minoryx.com

Active ingredient: Belumosudil For the treatment of chronic graft-versus-host disease (cGvHD) in adult patients after failure of at least two prior lines of systemic therapy 11.04.2024 11.04.2025

Responsible Person:
Sanofi-Aventis Germany GmbH
Lützowstrasse 107
10785 Berlin

Contact details:
Medical information from Sanofi
medinfo.de@sanofi.com
Phone: 0800 52 52 010
https://www.sanofi.de/de/kontakt

Active ingredient: Dordaviprone Treatment of H3 K27M-mutant diffuse glioma 15.04.2024 15.04.2025

Responsible Person:
Chimerix INC
2505 Meridian Parkway, Suite 100
NC 27713 Durham
USA

Contact details:
Managed Access Program Lead
Email: cup@chimerix.com

Active ingredient: Vorasidenib

Treatment of predominantly non-enhancing IDH-mutated oligodendrogliomas or astrocytomas in patients aged 12 years and older

(Treatment of predominantly non-enhancing IDH-mutated oligodendrogliomas or astrocytomas in patients aged 12 years and older)

22.05.2024 22.05.2025

Responsible Person:
Serve medical affairs
35 Rue de Verdun
92284 Suresnes Cedex
France

Contact details:
Dr. Anke Klein
Email: medinfo-de@servier.com