14-05-2024
The Russian Ministry of Health has registered domestic generic of Spinraza (nusinersen), an expensive drug used for the treatment of all types of spinal muscular atrophy (SMA) in children and adults, reports The Pharma Letter’s local correspondent.
14-05-2024
Chinese biopharma company Everest Medicines announced that Nefecon, a delayed release capsule of budesonide, has been successfully launched in China with the first prescription issued.
Asia PacificCalliditas TherapeuticsChinaEverest MedicinesFocus OnNefeconNephrology and HepatologyOne to Watch CompaniesPharmaceuticalProduct LaunchRare diseasesSweden
13-05-2024
US rare diseases focused biotech Fulcrum Therapeutics has entered into a collaboration and license agreement with French pharma major Sanofi for the development and commercialization of losmapimod.
BiotechnologyDealsFocus OnFranceFulcrum TherapeuticsLicensinglosmapimodMusculoskeletalOne to Watch CompaniesRare diseasesResearchSanofiUSA
13-05-2024
USA-based biotech Ajax Therapeutics today announced the completion of an oversubscribed $95 million Series C financing, led by Goldman Sachs with participation from new investors, including Eli Lilly.
AJ1-11095Ajax TherapeuticsBiotechnologyFocus OnHematologyMarkets & MarketingOne to Watch CompaniesRare diseasesRegulationUS FDAUSA
13-05-2024
Huntington's disease (HD) is a progressive neurodegenerative disorder characterized by involuntary movements, cognitive decline, and emotional disturbances.1 Recent global prevalence data cites 4.88 per 100,000 persons and an incidence of 0.48 cases per 100,000 person-years.2
AMT-130Drug TrialFocus OnFrom our correspondentHuntington's diseaseIn DepthIngrezzaNeurocrine BiosciencesPharmaceuticalpridopidinePrilenia TherapeuticsRare diseasesResearchSkyhawk TherapeuticsuniQure
11-05-2024
Privately-held Californian company Maze Therapeutics has found a new partner for its investigational Pompe disease candidate in the form of Japan’s Shionogi.
Asia PacificBiotechnologyDealsFocus OnGeneticsJapanLicensingMaze TherapeuticsMZE001One to Watch CompaniesPompeRare diseasesResearchShionogiUSA
10-05-2024
Italian drugmaker Recordati has recorded net revenues of 608 million euros ($650 million) in the first quarter of 2024, up 10.2%.
FinancialIsturisaItalyManagementPharmaceuticalRare diseasesRecordatiResearch
10-05-2024
US biotech bluebird bio saw its shares close up almost 15% at $1.12 yesterday, after it reported first quarter 2024 results and business highlights, including recent commercial and operational progress.
Biotechnologybluebird bioCell and Gene TherapyFinancialLyfgeniaManagementRare diseasesSkysonaUSAZynteglo
09-05-2024
New York-based biotech Regeneron Pharmaceuticals has announced highly encouraging results from a trial of its investigational gene therapy DB-OTO.
AudiologyBiotechnologyCell and Gene TherapyDB-OTODrug TrialRare diseasesRegeneron PharmaceuticalsResearchUSA
08-05-2024
Swedish Orphan Biovitrum has announced that the European Commission (EC) has approved an indication extension for Aspaveli (pegcetacoplan) to treat adults with paroxysmal nocturnal haemoglobinuria (PNH) who have hemolytic anemia.
AspaveliBiotechnologyEmpaveliEuropeEuropean Medicines AgencyFocus OnHematologyRare diseasesRegulationSwedenSwedish Orphan BiovitrumUSA
08-05-2024
US pharma giant Pfizer shared some sad clinical trial news Tuesday.
BiotechnologyCell and Gene TherapyDrug Trialfordadistrogene movaparvovecMusculoskeletalPfizerRare diseasesResearchUSA
08-05-2024
Final results from a three-year open-label extension study of Fintepla (fenfluramine) have been released by Belgium’s UCB.
BelgiumConferencesDrug TrialFinteplaGeneticsNeurologicalPharmaceuticalRare diseasesResearchUCB
07-05-2024
US biopharma Vertex Pharmaceuticals remains an evergreen pick in the stock market, announcing another set of financial results that exceeded expectations.
AnalgesiaCasgevyCell and Gene TherapyFinancialManagementPharmaceuticalRare diseasesRespiratory and PulmonaryUSAVertex Pharmaceuticals
07-05-2024
A trial flop for Massachusetts, USA-based EyePoint Pharmaceuticals has triggered a sell-off from investors, with the price of a share dropping by around half.
Drug TrialDuravyuEyePoint PharmaceuticalsOne to Watch CompaniesOphthalmicsPharmaceuticalRare diseasesResearchUSA
06-05-2024
AstraZeneca on Monday announced the successful completion of an equity investment with France-based Cellectis, a clinical-stage biotechnology company.
AstraZenecaBiotechnologyCell and Gene TherapyCellectis S.A.DealsImmunologicalsOne to Watch CompaniesRare diseasesUKVC & Investment
05-05-2024
The prestigious New England Journal of Medicine (NEJM) has published interim results from the Phase III trial of Japanese drug major Takeda Pharma’s Adzynma (apadamtase alfa/cinaxadamtase alfa; code name TAK-755) in patients with congenital thrombotic thrombocytopenic purpura (cTTP).
AdzynmaAsia PacificBiotechnologyDrug TrialFocus OnHematologyJapanRare diseasesResearchTAK-755Takeda
04-05-2024
Sentynl, the USA-based wholly-owned subsidiary of Indian generics major Zydus Lifesciences, has announced the closing of the sale of Eiger BioPharmaceuticals’ Zokinvy (lonafarnib) program to Sentynl.
DealsEiger BioPharmaceuticalsIndiaPharmaceuticalRare diseasesSentynll TherapeuticsUSAZokinvyZydus Lifesciences
02-05-2024
mRNA specialist Moderna has opted to end its collaboration with fellow US biotech Metagenomi on primary hyperoxaluria type 1 (PH1).
BiotechnologyCell and Gene TherapyDealsFocus OnGeneticsManagementMetagenomiModernaNephrology and HepatologyOne to Watch CompaniesRare diseasesUSA
30-04-2024
Shares of US developer of rare neurological drugs Harmony Biosciences (Nasdaq: HRMY) rose more than 10% to $32.30 today, after it announced the acquisition of Epygenix Therapeutics.
CNS DiseasesCompanies, mergers and acquisitionsEpygenix TherapeuticsHarmony BiosciencesNeurologicalOne to Watch CompaniesPharmaceuticalRare diseasesUSA
30-04-2024
USA-based Kedrion Biopharma has announced that Ryplazim (plasminogen, human-tvmh) is in stock and available for purchase in the USA.
BiotechnologyGeneticsKedrion BiopharmaProductionRare diseasesResearchRyplazimUSA
30-04-2024
X4 Pharmaceuticals has secured US approval for Xolremdi (mavorixafor), for the treatment of WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis).
Drug TrialOne to Watch CompaniesPharmaceuticalRare diseasesRegulationResearchUS FDAUSAX4 PharmaceuticalsXolremdi
30-04-2024
A full cycle of production of a drug for the treatment of Fabry disease will be launched in Russia this year, reports The Pharma Letter’s local correspondent.
FabagalFabrazymeFabryFocus OnFrom our correspondentGamaleyaIn DepthPetrovax FarmPharmaceuticalPricing, reimbursement and accessProductionRare diseasesRussiaRussian marketSanofi
29-04-2024
Family-controlled Spanish pharma company Esteve has signed a binding offer with USA-based Perrigo Company to acquire the latter’s HRA Pharma Rare Diseases, a specialist in rare and ultra-rare diseases.
Companies, mergers and acquisitionsKetoconazoleLaboratorios EsteveLysodrenManagementmetopironePerrigoPharmaceuticalRare diseasesSpainUSA
26-04-2024
Swedish Orphan Biovitrum, a rare disease company also known as Sobi, is set to raise around 10 billion Swedish kroner ($920 million) in debt.
BiotechnologyFinancialHematologyManagementRare diseasesResearchSwedenSwedish Orphan BiovitrumVC & InvestmentVonjo
25-04-2024
Sanofi shares were up by nearly 4% during Thursday’s early afternoon trading in Paris following its results presentation..
AltuviiioDupixentFinancialFranceImmunologicalsInflammatory diseasesManagementNexviadymeNexviazymeOncologyPharmaceuticalRare diseasesSanofi
25-04-2024
Shares in Britain’s largest drugmaker, AstraZeneca, have surged to a multi-month high following better-than-expected financial results for the first quarter.
AstraZenecaBiotechnologyCalquenceEnhertuFinancialImfinziImmunologicalsLynparzaManagementOncologyPharmaceuticalRare diseasesResearchTagrissoUK
A clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases.
