| RPI |
RPI |
92%
|
the Company sold to RPI a certain portion of the Company’s right to receive sales-based royalty payments |
| ROCHE |
ROCHE |
90%
|
Under the terms of the SMA License Agreement, Roche acquired an exclusive worldwide license to the Company’s SMA program. |
| HOSPITAL-AND-RETAIL-PHARMACIES |
HOSPITAL-AND-RETAIL-PHARMACIES |
80%
|
Our third-party distributors act as intermediaries between us and end-users and do not typically stock significant quantities of Translarna. |
| SMA-FOUNDATION |
SMA-FOUNDATION |
80%
|
In November 2011, the Company and the SMA Foundation entered into a licensing and collaboration agreement with Roche. |
| CLPT |
ClearPoint Neuro, Inc. |
72%
|
Reciprocal relationship from CLPT graph: Reciprocal relationship from PTCT graph: Reciprocal relationship from CLPT graph: Reciprocal relationship from PTCT graph: or its PTC-AADC indefinite-lived in… |
| CLPT |
ClearPoint Neuro, Inc. |
72%
|
or its PTC-AADC indefinite-lived intangible asset as of October 1, 2024 and recorded a partial impairment on the PTC-AADC indefinite-lived intangible asset of $ 159.5 million, which is recorded as in… |
| IONS |
Ionis Pharmaceuticals, Inc. |
72%
|
Reciprocal relationship from IONS graph: Reciprocal relationship from PTCT graph: Reciprocal relationship from IONS graph: Reciprocal relationship from PTCT graph: Reciprocal relationship from IONS g… |
| IONS |
Ionis Pharmaceuticals, Inc. |
72%
|
rmaceutical products for which marketing authorization has been granted are subject to extensive and rigorous regulation; ● Failure to obtain and maintain acceptable pricing and reimbursement terms f… |
| LXEO |
Lexeo Therapeutics, Inc. |
72%
|
Reciprocal relationship from LXEO graph: Reciprocal relationship from PTCT graph: Reciprocal relationship from LXEO graph: Reciprocal relationship from PTCT graph: Reciprocal relationship from LXEO g… |
| NVSEF |
Novartis AG |
72%
|
Reciprocal relationship from NVSEF graph: Reciprocal relationship from PTCT graph: Reciprocal relationship from NVSEF graph: Reciprocal relationship from PTCT graph: nion of the CHMP of the EMA to no… |
| NVSEF |
Novartis AG |
72%
|
nion of the CHMP of the EMA to not renew the authorization of Translarna for the treatment of nmDMD. This change in estimate for sales allowance resulted in an increase of $98.6 million to the sales … |
| RPRX |
Royalty Pharma plc |
72%
|
aspects of the Warren Lease dated as of May 24, 2022 between the Company and the Warren Landlord. Pursuant to the Warren Lease Amendment, as of the Surrender Effective Date (as defined in the Warren … |
| RPRX |
Royalty Pharma plc |
72%
|
Reciprocal relationship from RPRX graph: Reciprocal relationship from PTCT graph: aspects of the Warren Lease dated as of May 24, 2022 between the Company and the Warren Landlord. Pursuant to the War… |
| TRDA |
Entrada Therapeutics, Inc. |
72%
|
Reciprocal relationship from TRDA graph: everal risks, including disruptions in supply, price increases or late deliveries. There are, in general, relatively few alternative sources of supply for sub… |
| AGILIS-BIOTHERAPEUTICS-INC |
AGILIS-BIOTHERAPEUTICS-INC |
62%
|
ances as well as publicly available data for instruments with similar characteristics when calculating its incremental borrowing rates. The lease term for all of the Company’s leases includes the non… |
| AKCEA-THERAPEUTICS-INC |
AKCEA-THERAPEUTICS-INC |
62%
|
. If the EC adopts the negative opinion, Translarna would no longer have marketing authorization in the member states of the EEA. The marketing authorization for Translarna remains in effect, pending… |
| CATALYST-PHARMS-INC |
CATALYST-PHARMS-INC |
62%
|
imbursement to be provided for Emflaza are made on a plan-by-plan, and in some cases, on a patient-by-patient basis. Coverage and reimbursement decisions by third-party payors, including the processi… |
| CHEISI-FARMACEUTICA-INC |
CHEISI-FARMACEUTICA-INC |
62%
|
patients. Our ability to generate and realize revenue in Russia may be materially and adversely impacted as many countries, including the United States, have imposed and may continue to consider impo… |
| CHESI-FARMACEUTICA-INC |
CHESI-FARMACEUTICA-INC |
62%
|
patients. Our ability to generate and realize revenue in Russia may be materially and adversely impacted as many countries, including the United States, have imposed and may continue to consider impo… |
| COMPANY-AGILITY-MERGER-SUB-INC |
COMPANY-AGILITY-MERGER-SUB-INC |
62%
|
ances as well as publicly available data for instruments with similar characteristics when calculating its incremental borrowing rates. The lease term for all of the Company’s leases includes the non… |
| F-HOFFMAN-LA-ROCHE-LTD |
F-HOFFMAN-LA-ROCHE-LTD |
62%
|
finalize pricing and reimbursement negotiations with the applicable government body in such country. As a result, our commercial launch will continue to be on a country-by-country basis. We also have… |
| F-HOFFMAN-LA-ROCHE-LTD-HOFFMAN-LA-ROCHE-INC |
F-HOFFMAN-LA-ROCHE-LTD-HOFFMAN-LA-ROCHE-INC |
62%
|
es and discovery programs that are in early clinical, pre-clinical and research and development stages focused on the development of new treatments for multiple therapeutic areas for rare diseases. 3… |
| HOPEWELL-CAMPUS-OWNER-LLC |
HOPEWELL-CAMPUS-OWNER-LLC |
62%
|
aspects of the Warren Lease dated as of May 24, 2022 between the Company and the Warren Landlord. Pursuant to the Warren Lease Amendment, as of the Surrender Effective Date (as defined in the Warren … |
| MRI-INTERVENTIONS-INC |
MRI-INTERVENTIONS-INC |
62%
|
or its PTC-AADC indefinite-lived intangible asset as of October 1, 2024 and recorded a partial impairment on the PTC-AADC indefinite-lived intangible asset of $ 159.5 million, which is recorded as in… |
| NOVARTIS-PHARMACEUTICALS-CORPORATION |
NOVARTIS-PHARMACEUTICALS-CORPORATION |
62%
|
finalize pricing and reimbursement negotiations with the applicable government body in such country. As a result, our commercial launch will continue to be on a country-by-country basis. We also have… |
| RBC-CAPITAL-MARKETS-LLC |
RBC-CAPITAL-MARKETS-LLC |
62%
|
for vatiquinone for the treatment of children and adults living with FA. In February 2025, the FDA accepted for filing the NDA and granted priority review with a target regulatory action date of Augu… |
