FDA approves non-steroidal treatment for Duchenne muscular dystrophy

21 March 2024 - Today, the US FDA approved Duvyzat (givinostat) oral medication for the treatment of Duchenne muscular dystrophy in ...

Read more →

Phanes Therapeutics' PT886 granted fast track designation for the treatment of patients with metastatic claudin 18.2 positive pancreatic adenocarcinoma by the FDA

20 March 2024 - Phanes Therapeutics announced today that the US FDA has granted fast track designation to PT886 for the ...

Read more →

Spevigo approved for expanded indications in China and the US

19 March 2024 - The US FDA’s approval expands the use of Spevigo to include the treatment of generalised pustular psoriasis in ...

Read more →

A lifesaving therapy for children with a rare disease is now the world’s most expensive drug, raising questions about access

20 March 2024 - A new gene therapy for the fatal genetic disorder metachromatic leukodystrophy, or MLD, will carry a ...

Read more →

US FDA approves Idorsia’s once-daily Tryvio (aprocitentan) – the first and only endothelin receptor antagonist for the treatment of high blood pressure not adequately controlled in combination with other antihypertensives

20 March 2024 - Idorsia plans to make Tryvio available to the millions of patients in the US who are not ...

Read more →

PTC Therapeutics provides key regulatory updates (March 2024)

19 March 2024 - BLA submitted to FDA for Upstaza for the treatment of AADC deficiency - ...

Read more →

Deverra Therapeutics granted FDA regenerative medicine advanced therapy and orphan drug designations for DVX101 (Dilanubicel) for the treatment of acute myeloid leukaemia

19 March 2024 - Deverra Therapeutics today announced that the US FDA granted regenerative medicine advanced therapy designation to its lead ...

Read more →

FDA grants accelerated approval to ponatinib with chemotherapy for newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia

19 March 2024 - Today, the FDA granted accelerated approval to ponatinib (Iclusig, Takeda Pharmaceuticals) with chemotherapy for adult patients ...

Read more →

Citius Pharmaceuticals announces FDA acceptance of the BLA resubmission of Lymphir (denileukin diftitox) for the treatment of adults with relapsed or refractory cutaneous T-cell lymphoma

18 March 2024 - FDA assigns Prescription Drug User Fee Act target action date of 13 August 2024 ...

Read more →

FDA approves first gene therapy for children with metachromatic leukodystrophy

18 March 2024 - Today, the US FDA approved Lenmeldy (atidarsagene autotemcel), the first FDA approved gene therapy indicated for the ...

Read more →

Xhance approved by FDA as first and only medication indicated for treatment of adults with chronic rhinosinusitis without nasal polyps

15 March 2024 - ReOpen was the first ever large placebo-controlled clinical trial program to demonstrate statistically significant reduction of symptoms ...

Read more →

Zymfentra (infliximab-dyyb), the first and only FDA approved subcutaneous infliximab, now commercially available in the U.S.

17 March 2024 - Zymfentra is commercially available across the US on 15 March 2024. ...

Read more →

BeiGene receives FDA approval for Tevimbra for the treatment of advanced or metastatic oesophageal squamous cell carcinoma after prior chemotherapy

14 March 2024 - Results from the global, Phase 3 RATIONALE 302 trial showed Tevimbra prolonged the survival of patients who ...

Read more →

ImmVira's oncolytic product MVR-T3011 IT intratumoral injection receives FDA fast track designation for HNSCC treatment

15 March 2024 - ImmVira has recently announced that the US FDA has granted fast track designation for oncolytic virus product ...

Read more →

US FDA approves Bristol Myers Squibb’s Breyanzi as the first and only CAR T cell therapy for adults with relapsed or refractory chronic lymphocytic leukaemia or small lymphocytic lymphoma

14 March 2024 - In TRANSCEND CLL 004, the first pivotal multicenter trial to evaluate a CAR T cell therapy in ...

Read more →