Rare disease

Rinascera launches with two rare genetic skin disease therapies

Mon, 20 Jul 2026 12:00:00 -0400

Rinascera Therapeutics Inc. has secured enough initial funding to acquire two drugs for rare genetic skin diseases, despite stalled development with their previous owners. The company, whose name comes from the Latin root renasci – to be born again, was incubated by Olive Tree Capital, which led the seed round with participation from Civilization Ventures and Toba Capital.

Drug Farm bags $55M series D to advance AI-developed ALPK1 drug

Tue, 14 Jul 2026 12:00:00 -0400

Drug Farm Biotechnology Co. Ltd. closed a $55 million series D round to advance its AI-developed alpha-protein kinase 1 (ALPK1) inhibitor, DF-003, in a phase III trial for ROSAH syndrome, a rare genetic disease that can lead to blindness.

Opus aligns with FDA on rare eye disease gene therapy trial

Tue, 07 Jul 2026 12:00:00 -0400

Opus Genetics Inc. has secured U.S. FDA alignment on an eight-patient phase III trial of its lead gene therapy, OPGx-LCA5, for an ultra-rare form of inherited childhood blindness.

Endocrinology becomes Vertex’s fifth pillar with $10B Crinetics buy

Tue, 07 Jul 2026 12:00:00 -0400

Representing the fourth largest biopharma M&A announced in 2026, Vertex Pharmaceuticals Inc. offered $85 per share in cash, or about $10 billion, to buy endocrine specialist Crinetics Pharmaceuticals Inc., adding the acromegaly drug Palsonify and a phase III asset, atumelnant, for congenital adrenocorticotropic hyperplasia (CAH).

Vertex buying Crinetics for $10B

Mon, 06 Jul 2026 12:00:00 -0400

Vertex Pharmaceuticals Inc. is buying Crinetics Pharmaceuticals Inc. for $85 per share in cash, which translates to about $10 billion in equity value. Both boards approved the transaction, expected to close in the third quarter of 2026.

EMA to pull Amgen’s Tavneos over ‘incorrect and misleading’ data

Fri, 26 Jun 2026 12:00:00 -0400

The EMA is recommending withdrawing Amgen Inc.’s complement inhibitor Tavneos (avacopan) from the market in Europe, saying data provided at the time it assessed the MAA “were found to be incorrect and misleading and could no longer be relied upon for demonstrating Tavneos’ effectiveness.”

Another FDA no for Camurus’ monthly acromegaly drug Oclaiz

Thu, 11 Jun 2026 12:00:00 -0400

In a repeat move, the U.S. FDA issued yet again a complete response letter (CRL) to Camurus AB for its subcutaneous extended-release injection drug CAM-2029 (octreotide) to treat the rare chronic growth disorder acromegaly. The drug, which expects to be branded Oclaiz in the U.S. upon approval, is called Oczyesa in the EU and the U.K., where it received marketing authorization in 2025.

Travere expands rare kidney portfolio in $1B+ Everest deal

Tue, 02 Jun 2026 12:00:00 -0400

Less than two months after winning FDA approval for a second indication for Filspari (sparsentan), Travere Therapeutics Inc. added to its rare kidney disease pipeline by exclusively licensing civorebrutinib from Everest Medicines Ltd. in a deal that could be worth more than $1.14 billion.

Elzonris this: FDA clears Abbvie’s Decnupaz in BPDCN

Thu, 28 May 2026 12:00:00 -0400

Payoff for the November 2023 buyout of Immunogen Inc. came for Abbvie Inc. in the form of U.S. FDA clearance for the CD123-targeting antibody-drug conjugate Decnupaz (pivekimab sunirine) to treat adults with blastic plasmacytoid dendritic cell neoplasm (BPDCN), an ultra-rare, aggressive and quick-killing hematologic malignancy.

China’s new IIT rules expected to wipe out ‘gray market’

Tue, 26 May 2026 11:00:00 -0400

New regulations tighten regulatory oversight of China’s investigator-initiated trials (IITs) but legitimize the pathway that will be open to other modalities beyond cell and gene therapies.

China’s new IIT rules expected to wipe out ‘gray market’

Fri, 22 May 2026 12:00:00 -0400

New regulations tighten regulatory oversight of China’s investigator-initiated trials (IITs) but legitimize the pathway that will be open to other modalities beyond cell and gene therapies.

Relay’s zovegalisib hits in phase II vascular anomalies trial

Tue, 19 May 2026 12:00:00 -0400

Showing a significant efficacy signal in a phase II trial, Relay Therapeutics Inc.’s zovegalisib (RLY-2608) achieved a 60% volumetric response in patients with PIK3CA-driven vascular anomalies (VAs). The isoform-selective PI3Ka inhibitor is in late-stage clinical trials with various combinations for P13Ka-mutated, HR+/HER2- advanced breast cancer, with VAs representing a second indication for which Leerink Partners analyst Andrew Berens forecasts $2.8 billion in peak revenues.

EU reaches Critical Medicines Act pact to tackle drug shortages

Fri, 15 May 2026 12:00:00 -0400

In further fallout from the COVID-19 pandemic, the EU has now reached agreement on the Critical Medicines Act, which aims to boost European manufacturing capacity for drugs and their active ingredients, and enable member states to organize joint procurement to improve access to orphan drugs.

Italy’s Angelini pays $4.1B cash for rare disease specialist Catalyst

Thu, 07 May 2026 12:00:00 -0400

Italian family-owned Angelini Pharma SpA is making its first move into the U.S. market, acquiring rare diseases specialist Catalyst Pharma Inc. in an all-cash deal worth $4.1 billion. The acquisition gives Angelini ownership of three marketed drugs for treating epilepsy and neuromuscular diseases that had combined sales of $589 million in 2025, a 19.8% increase over 2024.

Up to BAT in PSC, Mirum base case proven with phase IIb

Mon, 04 May 2026 12:00:00 -0400

Entering what Mirum Pharmaceuticals Inc. CEO Chris Peetz called “a new phase of growth and value creation,” the company plans to submit an NDA to the U.S. FDA in the second half of this year, based on phase IIb data from the Vistas trial with oral ileal bile acid transporter (IBAT) inhibitor volixibat in primary sclerosing cholangitis (PSC).

Lilly buying Ajax for up to $2.3B for next-gen JAK assets

Mon, 27 Apr 2026 12:00:00 -0400

Eli Lilly and Co. is acquiring Ajax Therapeutics Inc. for up to $2.3 billion in cash, gaining access to next-generation JAK inhibitors for patients with myeloproliferative neoplasms.

Travere’s Filspari approved for rare kidney disease FSGS

Tue, 14 Apr 2026 08:00:00 -0400

Becoming the first and only fully FDA-approved treatment for focal segmental glomerulosclerosis (FSGS), Travere Therapeutics Inc.’s Filspari (sparsentan) has gained access to a second lucrative market with a regulatory package that validates proteinuria as a surrogate endpoint.

Synox en route to approval on positive phase III for TGCT

Mon, 13 Apr 2026 12:00:00 -0400

Synox Therapeutics Ltd. is preparing to file for FDA approval of emactuzumab in treating tenosynovial giant cell tumor (TGCT), after announcing positive top-line phase III results.

Pediatric PRVs top the voucher popularity contest

Thu, 09 Apr 2026 12:00:00 -0400

With three rare pediatric disease priority review vouchers (RPD PRVs) awarded just since the end of March, the nearly year-and-a-half lapse in the program’s reauthorization seems to have had little short-term impact. The three new vouchers bring the total RPD PRVs granted so far this year to seven – one more than the agency issued all last year and down two from the nine given in 2024.

Vykat caveat: Neurocrine’s $2.9B Soleno bid low-ball?

Mon, 06 Apr 2026 12:00:00 -0400

A proposal to buy out Soleno Therapeutics Inc. didn’t wait for European approval of Vykat XR (diazoxide choline) to treat hyperphagia in Prader-Willi syndrome, as Neurocrine Biosciences Inc. is putting on the table $53 per share in cash, which equates to an equity value of $2.9 billion.

LHON updates: Gene therapy progress; idebenone receives US CRL

Thu, 02 Apr 2026 12:00:00 -0400

Chiesi Group’s idebenone faced a regulatory setback last month after the U.S. FDA issued a complete response letter (CRL) to the company’s NDA for Leber hereditary optic neuropathy (LHON), a rare inherited disorder that causes sudden vision loss.But a chance missed for Chiesi may be an opportunity for gene therapies, including Gensight Biologics SA’s lenadogene nolparvovec (Lumevoq; GS-010).

FDA approves Rocket’s Kresladi as first gene therapy for LAD-I

Fri, 27 Mar 2026 12:00:00 -0400

In a win for the rare disease space, the U.S. FDA granted accelerated approval for Rocket Pharmaceuticals Inc.’s Kresladi (marnetegragene autotemcel) as the first gene therapy option for treating severe leukocyte adhesion deficiency-I (LAD-I), an ultrarare genetic immune disorder characterized by an immunodeficiency predisposing those affected to recurrent and fatal infections.

GSK’s IBAT inhibitor wins US FDA nod for cholestatic pruritus

Thu, 19 Mar 2026 12:00:00 -0400

With the U.S. FDA’s approval of GSK plc’s ileal bile acid transporter (IBAT) inhibitor, Lynavoy (linerixibat), patients with primary biliary cholangitis no longer need off-label treatments for a debilitating internal itch symptom called cholestatic pruritus.

Pharma USA: The complex journey of launching rare disease drugs

Tue, 17 Mar 2026 12:00:00 -0400

Rare disease drug development companies battle with economic challenges and small patient populations, but new technology alongside a human connection are helping researchers and marketers identify patients, educate physicians and build networks. At Pharma USA, a Reuters Events meeting in Philadelphia March 16-17, executives from Astrazeneca plc and UCB SA discussed their successes with rare disease launches, presenting their playbooks for building trust and driving adoption among patients.

Forecast brighter for Uniqure with Prasad’s departure?

Mon, 09 Mar 2026 12:00:00 -0400

The regulatory clouds that have been darkening the U.S. FDA landscape of late for Uniqure NV’s gene therapy AMT-130 in Huntington’s disease may be parting a bit with the announced departure of Vinay Prasad as director of the agency’s CBER at the end of April.

FDA’s rare disease toolbox not fully used

Wed, 04 Mar 2026 12:00:00 -0500

At the current pace of innovation in the U.S. rare disease space, developing and approving therapies for just half of the 10,000-plus known rare diseases would take more than 160 years, Bradley Campbell, president and CEO of Amicus Therapeutics Inc., recently told the Senate Committee on Aging.

Experts: Regulatory roadblocks stalling rare disease therapies

Tue, 03 Mar 2026 12:00:00 -0500

A lot of distance lies between talking regulatory flexibility and actually being flexible. That message was driven home again after Uniqure NV disclosed in its latest earnings report March 2 that the U.S. FDA wants a sham-controlled study before it will consider approval of the company’s gene therapy AMT-130 in Huntington’s, a rare disease currently affecting about 41,000 people in the U.S.

Theravance slashes R&D as hypotension asset fails again

Tue, 03 Mar 2026 12:00:00 -0500

Another phase III failure of Theravance Biopharma Inc.’s norepinephrine reuptake inhibitor ampreloxetine to treat symptomatic neurogenic orthostatic hypotension, this time in patients with the rare disease multiple system atrophy, means the end of the company’s R&D efforts. Cutting its workforce in half and terminating all development of ampreloxetine, its only pipeline product, the Dublin-based company’s shares (NASDAQ:TBPH) sank by 26%, or $4.99, to close March 3 at $13.96.

State of the biopharma industry filled with uncertainty

Wed, 25 Feb 2026 12:00:00 -0500

While the annual State of the Union address has morphed over the years from a summation of the state of the U.S. government and the president’s legislative agenda into political theater on both sides of the aisle, President Donald Trump did include some recommendations to Congress in his Feb. 24 speech. Among those recommendations was a request for Congress to codify his most-favored-nation pricing policy for prescription drugs.

New FDA guidance details ‘plausible mechanism’ pathway

Mon, 23 Feb 2026 12:00:00 -0500

The U.S. FDA rolled out its anticipated “plausible mechanism” draft guidance, enabling ultrarare disease drug developers to generate evidence of effectiveness and safety to support approval when randomized controlled trials are not possible due to small patient populations.