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    NEWS

    DRUGS & DEVELOPMENTS

    MARKET INTEL by PharmaCompass

    Mission CDMO team brings reliability, resourcefulness, & responsiveness in executing your development project.
    All News #Library
    Rare Diseases

    FDA Approves First Gene Therapy for Rare Immune Disorder

    REUTERS

    FDA Grants Breakthrough Designation for Adrabetadex Infantile NPC

    BUSINESSWIRE

    Canada Approves Biogen`s ZURZUVAE for Postpartum Depression

    PHARMIWEB

    Recordati Rare Diseases Presents Long-Term Safety Data

    PR NEWSWIRE

    Saol Therapeutics Secures FDA Type A Meeting For SL1009

    PR NEWSWIRE

    Satellos Secures Clearance for SAT-3247 in DMD Phase 2 Trial

    PHARMIWEB

    Agios Provides Update on U.S. sNDA for Mitapivat in Thalassemia

    GLOBENEWSWIRE

    Dyne readies FDA push after DMD exon 51 med excels in trial

    FIERCE BIOTECH

    Merck mulls PV program’s future while Takeda takes victory lap

    FIERCE BIOTECH

    Mirum Pharma Enters into Definitive Agreement to Acquire Bluejay

    PHARMIWEB

    Results for UCB`s Fintepla pave way for 3rd epileptic indication

    FIERCE PHARMA

    Dyne Therapeutics To Review Duchenne Muscular Dystrophy Trial

    GLOBENEWSWIRE

    Lundbeck Reveals Bexicaserin`s Positive Long-Term Phase 2 Data

    PR NEWSWIRE

    BMS`s Breyanzi Is First CAR-T Approved in US for Rare Lymphoma

    BUSINESSWIRE

    Jazz Pharma Spotlights Epidiolex for Refractory Epilepsy at AES

    PR NEWSWIRE

    FDA Approves Omisirge(R) For Severe Aplastic Anemia

    PHARMIWEB

    Sobi Highlights Haematology Commitment At ASH 2025

    PR NEWSWIRE

    Soligenix Advancing Rare-Disease Positioned at Pivotal Crossroad

    GLOBENEWSWIRE

    Stoke, Biogen Unveil Zorevunersen Data Syndrome for Dravet at AES

    PHARMIWEB

    Vicore To Present At Oppenheimer Rare Disease Summit

    PHARMIWEB

    After 20 Years, Medtronic Claims Neurostim Approval In Dystonia

    FIERCE BIOTECH

    VALIANT Pharma Reports Positive Phase 3 Results

    PHARMIWEB

    Breakthrough Therapies Rise as Rare Diseases Burden Aging

    GLOBENEWSWIRE

    ALS Association Invests $2M In Early-Stage ALS Therapy Trials

    PR NEWSWIRE

    J & D Pharma Gets Orphan Drug Status For Hepatocellular Carcinoma

    PR NEWSWIRE

    Bayer Initiates Phase 2 Trial For Alport Syndrome Treatment

    PHARMIWEB

    Denali Ph. 1 Pompe Plans Hit With Clinical Hold

    FIERCE BIOTECH

    Capricor Eyes Nod for Duchenne Therapy After Success in Study

    GLOBENEWSWIRE

    NEJM Publishes VALIANT EMPAVELI Results for C3G & Primary IC-MPGN

    GLOBENEWSWIRE

    GRIN Therapeutics Showcases Global Phase 3 Radiprodil Trial

    PR NEWSWIRE

    Pharvaris Reports Deucrictibant`s Efficacy In HAE Attack

    GLOBENEWSWIRE

    Soligenix`s Innovative Platform Revolutionizes Treatment for CTCL

    GLOBENEWSWIRE

    Mezzion Bolsters Udenafil Exclusivity In Fontan Patients

    PR NEWSWIRE

    House Vote Moves FDA`s Rare PRV Program A Step Closer To Revival

    FIERCE PHARMA

    Belite Brings FDA Filing Into View with Phase 3 Stargardt Win

    PRESS RELEASE

    Akebia Announces Establishment Of Rare Kidney Disease Pipeline

    GLOBENEWSWIRE

    Biocodex To Unveil New Data At AES 2025 Meeting

    PR NEWSWIRE

    Biodexa Enrolls First EU Patients in Pivotal Serenta FAP Trial

    GLOBENEWSWIRE

    KALA BIO Secures $6M Investment From Investor David E. Lazar

    GLOBENEWSWIRE

    Mirum Pharma Enrolls First Patient In BLOOM Ph2 Study

    BUSINESSWIRE

    NanoViricides Inks MSA for NV-387 Orphan Drug Designation

    PHARMIWEB

    Solid Biosciences Gets FDA Rare Pediatric Disease Designation

    GLOBENEWSWIRE

    Sound Pharma Gets FDA Breakthrough Status For SPI-1005

    PHARMIWEB

    Upsher-Smith Launches Kymbee for Duchenne Muscular Dystrophy

    PR NEWSWIRE

    FDA Grants Medivir Orphan Drug Designation For MIV-711

    PHARMIWEB

    EMA Grants Orphan Status To Kedrion`s Aceruloplasminemia

    PHARMIWEB

    FDA Delays Decision On Ascendis` Dwarfism Prospect By 3 Months

    PRESS RELEASE

    Cure SMA Welcomes FDA Approval of Itvisma for SMA

    PR NEWSWIRE

    Renalys Pharma Reports Positive Phase 3 Sparsentan Results

    PR NEWSWIRE

    FDA Extends Review of TransCon CNP for Pediatric Achondroplasia

    GLOBENEWSWIRE

    Commenters Seek FDA Orphan Status for Disseminated Cocci Drugs

    RAPS

    Chemomab Joins Oppenheimer`s Rare Disease Summit

    GLOBENEWSWIRE

    EMD Serono Innovates Rare Neuromuscular Disorder Treatment

    BUSINESSWIRE

    Jaguar Health Strengthens Global IP With New Crofelemer Patent

    PHARMIWEB

    Novartis Gets FDA Approval For Itvisma Gene Therapy In SMA

    PR NEWSWIRE

    Sarepta Updates SRP-1003 for Type 1 Myotonic Dystrophy

    BUSINESSWIRE

    Ultragenyx Issues Inducement Grant Per Nasdaq LR 5635(C)(4)

    GLOBENEWSWIRE

    Oxford-Harrington Rare Disease Centre Unveils 2025 Scholars

    PR NEWSWIRE

    Crinetics Initiates Ph 3 For Paltusotine in Carcinoid Syndrome

    GLOBENEWSWIRE

    Agios’ Pyrukynd turns in mixed results in sickle cell ph. 3

    GLOBENEWSWIRE

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